Glutamine Combined With Thalidomide in Preventing Radiation-induced Oral Mucositis in Patients Undergoing radiotherapy-a Multicenter, Open-label, Randomized Controlled Study

The goal of this study is to compare glutamine combined with thalidomide with glutamine alone in preventing radiation-induced oral mucositis. The aim of this study is to answer whether glutamine plus thalidomide could improve the median incidence time of grade 2 oral mucositis. Participants would be randomly divided into the two groups above mentioned.

Start Date15 Sep 2023

Sponsor / Collaborator-

CTRI/2023/08/057147 / Not yet recruitingPhase 3IIT

The role of glutamine in prevention of chemotherapy- induced peripheral neuropathy in breast cancer patients - NIL

Start Date05 Sep 2023

Sponsor / Collaborator

Jawaharlal Nehru University

100 Clinical Results associated with L-Glutamine

100 Translational Medicine associated with L-Glutamine

100 Patents (Medical) associated with L-Glutamine

6,218

Literatures (Medical) associated with L-Glutamine

31 Dec 2024·Journal of the International Society of Sports Nutrition

Supplementation of L-glutamine enhanced mucosal immunity and improved hormonal status of combat-sport athletes

Article

Author: Zheng, Ai-Chi ; Lu, Tung-Lin ; Fang, Shih-Hua ; Lu, Chi-Cheng ; Suzuki, Katsuhiko ; Wang, Chung-Yuan

BACKGROUND:

Maintaining proper immune function and hormone status is important for athletes to avoid upper respiratory tract infection (URTI) and insufficient recovery, which is detrimental to sport performance and health. The aim of this study was to evaluate whether three-week supplementation of L-glutamine could benefit the mucosal immunity and hormonal status of combat-sport athletes as well as their rates of upper respiratory tract infection (URTI) and subjective feelings of well-being after intensive training.

METHODS:

Twenty-one combat-sport athletes from the National Taiwan University of Sport were recruited in this study. After intensive training, two groups of the participants were asked to consume powder form of 0.3 g/kg body weight of L-glutamine (GLU group) or maltodextrin (PLA group) with drinking water in a randomized design at the same time every day during 3 weeks. Saliva samples were collected to measure immunoglobulin A (IgA), nitric oxide (NO), testosterone (T) and cortisol (C) before and after three-week supplementation; moreover, Hooper's index questionnaires were completed for wellness assessment. The incidence and duration of URTI were recorded by using a health checklist throughout the entire study period.

RESULTS:

Supplementation of L-glutamine significantly enhanced the concentrations of IgA and NO in saliva; additionally, the incidence of URTI was significantly reduced. Regarding hormones, T concentration was significantly decreased in the PLA group, whereas C concentration was significantly increased, resulting in a significant decrease of T/C ratio. In contrast, the GLU group showed a significant increase of T/C ratio, while the mood scores of the Hooper's index questionnaire were higher in the PLA group.

CONCLUSIONS:

Three-week supplementation of L-glutamine after intensive training enhanced the mucosal immunity, improved hormonal status and reduced the rate of URTI of combat-sport athletes while feelings of well-being were also enhanced. Therefore, L-glutamine would be beneficial for the sports performance and recovery of athletes.

01 Mar 2024·International journal of biological macromolecules

Global Lrp regulator protein from Haloferax mediterranei: Transcriptional analysis and structural characterization

Article

Author: Camacho, Mónica ; Bonete, María-José ; García-Bonete, María-José ; Fillat, María F ; Matarredona, Laura ; Esclapez, Julia ; Guío, Jorge

Haloferax mediterranei, an extreme halophilic archaeon thriving in hypersaline environments, has acquired significant attention in biotechnological and biochemical research due to its remarkable ability to flourish in extreme salinity conditions. Transcription factors, essential in regulating diverse cellular processes, have become focal points in understanding its adaptability. This study delves into the role of the Lrp transcription factor, exploring its modulation of glnA, nasABC, and lrp gene promoters in vivo through β-galactosidase assays. Remarkably, our findings propose Lrp as the pioneering transcriptional regulator of nitrogen metabolism identified in a haloarchaeon. This study suggests its potential role in activating or repressing assimilatory pathway enzymes (GlnA and NasA). The interaction between Lrp and these promoters is analyzed using Electrophoretic Mobility Shift Assay and Differential Scanning Fluorimetry, highlighting l-glutamine's indispensable role in stabilizing the Lrp-DNA complex. Our research uncovers that halophilic Lrp forms octameric structures in the presence of l-glutamine. The study reveals the three-dimensional structure of the Lrp as a homodimer using X-ray crystallography, confirming this state in solution by Small-Angle X-ray Scattering. These findings illuminate the complex molecular mechanisms driving Hfx. mediterranei's nitrogen metabolism, offering valuable insights about its gene expression regulation and enriching our comprehension of extremophile biology.

01 Mar 2024·CNS & neurological disorders drug targets

Nutritional Support System (NSS) as a New Therapeutic Strategy for Cerebral Palsy

Article

Author: Ibarra, Antonio ; Leal-Martinez, Fernando ; Jimenez Ramirez, Guadalupe

Abstract::

Cerebral palsy (CP) is part of a group of nonprogressive motor disorders. The disease affects movement and posture and constitutes the most frequent cause of motor disability in childhood. CP is characterized by spasticity, reflecting lesions in the pyramidal pathway. Treatment is currently focused on physical rehabilitation, and the annual progression of the disease is 2-3%. About 60% of these patients present severe degrees of malnutrition associated with dysphagia, gastrointestinal abnormalities, malabsorption, increased metabolism, and depression. These alterations promote sarcopenia functional dependence and affect the quality of life and delay the evolution of motor skills. Currently, there is evidence that the supplementation of several nutrients, dietary correction, and probiotics can improve neurological response by stimulating neuroplasticity, neuroregeneration, neurogenesis, and myelination. This therapeutic strategy could shorten the response period to treatment and increase both gross and fine motor skills. The interaction of nutrients and functional foods integrating a Nutritional Support System (NSS) has shown greater efficiency in neurological stimulation than when nutrients are supplied separately. The most studied elements in the neurological response are glutamine, arginine, zinc, selenium, cholecalciferol, nicotinic acid, thiamine, pyridoxine, folate, cobalamin, Spirulina, omega-3 fatty acids, ascorbic acid, glycine, tryptophan, and probiotics. The NSS represents a therapeutic alternative that will restore neurological function in patients with spasticity and pyramidal pathway lesions, both characteristics of patients with CP.

News (Medical) associated with L-Glutamine

14 Jun 2024

·www.biospace.com

Vertex Presents Positive Long-Term Data On CASGEVY™ (exagamglogene autotemcel) at the 2024 Annual European Hematology Association (EHA) Congress

Results from CLIMB-111, -121 and -131 accepted for oral presentation Data from these trials, with the longest follow-up of more than five years, demonstrate transformative, consistent and durable benefit of CASGEVY™ Safety pro with busulfan conditioning and autologous hematopoietic stem cell transplant BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced longer-term data for CASGEVY™ (exagamglogene autotemcel [exa-cel]) from global clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the annual European Hematology Association (EHA) Congress, confirm the transformative, consistent and durable clinical benefits of CASGEVY over time. CASGEVY is the first and only approved CRISPR-based gene-editing therapy. The data being presented are from more than 100 patients (46 SCD; 56 TDT) treated with exa-cel in clinical trials, with the longest follow-up now extending more than 5 years. The efficacy results are consistent with the previously reported primary and key secondary endpoints analyses from these exa-cel studies and continue to demonstrate transformative clinical benefit with durable and stable levels of fetal hemoglobin (HbF) and allelic editing. “The transformative benefit seen in patients with sickle cell disease in the trial is impressive given the significant and cumulative burden of disease faced by people living with this blood disorder,” said Haydar Frangoul, M.D., M.S., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute and HCA Healthcare’s TriStar Centennial Children’s Hospital. “I am eager to offer this therapy and the opportunity of a potential functional cure to my eligible patients.” “The comprehensive data set presented today for adult and adolescent TDT patients adds to the growing body of evidence for CASGEVY, and it is important to now ensure the availability of this innovative treatment to patients in the real world as soon as possible,” said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Gesù Children’s Hospital. “With the longest follow up now more than five years, alongside stable editing and sustained fetal hemoglobin levels, I have conviction in the durable benefit to the patients treated with CASGEVY.” New data presented from CASGEVY pivotal trials In SCD 36/39 (92.3%) evaluable patients (those with at least 16 months of follow-up) were free from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12), consistent with the previously reported primary endpoint data. Mean duration of VOC-free was 27.9 months, with a maximum of 54.8 months. 38/39 (97.4%) patients with at least 16 months of follow-up were free from hospitalizations related to VOCs for at least 12 consecutive months (HF12), consistent with the previously reported key secondary endpoint data. In TDT 49/52 (94.2%) evaluable patients (those with at least 16 months of follow-up) were transfusion-independent for at least 12 consecutive months with a mean weighted hemoglobin of at least 9 g/dL (TI12), consistent with the previously reported primary endpoint data. Mean duration of transfusion independence was 31.0 months, with a maximum of 59.4 months. All TDT patients dosed with at least 16 months of follow up are transfusion free. Two of the three patients who did not achieve TI12 in CLIMB-111 achieved TI12 in the long-term follow-up study, CLIMB-131, and have been transfusion independent for over one year. The third has been transfusion free for 3.4 months. Both SCD and TDT patients reported sustained and clinically meaningful improvements in their quality of life, including physical, emotional, social/family and functional well-being, and overall health status. In both SCD and TDT patients, edited levels of BCL11A alleles were stable over time in bone marrow and peripheral blood indicating successful editing in the long-term hematopoietic stem cells. All patients engrafted neutrophils and platelets after exa-cel infusion. The safety pro exa-cel was generally consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant. These longer-term data for CASGEVY from the CLIMB clinical trials will be shared as outlined below: Abstracts S273 and S274 will be oral presentations entitled “Exagamglogene Autotemcel For Severe Sickle Cell Disease,” and “Exagamglogene Autotemcel For Transfusion-Dependent Beta-Thalassemia,” on Sunday, June 16 at 12:15 CEST and 12:30 CEST, respectively. Abstracts P1493 and P1525 will be poster presentations entitled “Health-Related Quality Of Life Improvements After Exagamglogene Autotemcel In Patients With Severe Sickle Cell Disease,” and “Health-Related Quality Of Life Improvements After Exagamglogene Autotemcel In Patients With Transfusion-Dependent Βeta-Thalassemia,” on Friday, June 14 at 18:00 CEST. These presentations will include updated pivotal trial data from patients treated with CASGEVY in CLIMB-111 and CLIMB-121 and followed in CLIMB-131. Vertex will also share five health economics abstracts at the EHA Congress. Abstract P1483 is entitled “Adherence, Treatment Use, and Clinical Outcomes in Patients With Sickle Cell Disease With Recurrent Vaso-Occlusive Crises Treated With L-Glutamine, Voxelotor, or Crizanlizumab in the United States.” Abstract P1506 is entitled “Mortality and Clinical Complications Among Patients with Sickle Cell Disease With Recurrent VOCs in Canada.” Abstract P1507 is entitled “Treatment Utilization and Clinical Complications in Patients with Sickle Cell Disease Receiving Frequent Red Blood Cell Transfusions in the United States.” Abstract P2191 is entitled “Clinical Complications and Treatment Use Among Patients With Sickle Cell Disease With Recurrent Vaso-Occlusive Crises in the Netherlands.” Abstract PB3248 is entitled “Clinical Complications Among Patients With Transfusion-Dependent Beta-Thalassemia in the Netherlands.” About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with SCD is around 40 years. Stem cell transplant from a matched donor is a potentially curative option but is only available to a small fraction of people living with SCD because of the lack of available donors. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and significant health care resource utilization. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In Europe, the mean age of death for patients living with TDT is 50-55 years. Stem cell transplant from a matched donor is a potentially curative option but is only available to a small fraction of people living with TDT because of the lack of available donors. About CASGEVY™ (exagamglogene autotemcel [exa-cel]) CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients. About the CLIMB Studies The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD, characterized by recurrent VOCs, respectively. The trials are now closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the safety and efficacy of CASGEVY in patients who received CASGEVY in other CLIMB studies. The trial is designed to follow patients for up to 15 years after CASGEVY infusion. U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY (exagamglogene autotemcel) WHAT IS CASGEVY? CASGEVY is a one-time therapy used to treat people aged 12 years and older with: sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs beta thalassemia (β-thalassemia) who need regular blood transfusions CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia. IMPORTANT SAFETY INFORMATION What is the most important information I should know about CASGEVY? After treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels. Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of platelet cells: severe headache abnormal bruising prolonged bleeding bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood Tell your healthcare provider right away if you experience any of the following, which could be signs of low levels of white blood cells: fever chills infections You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects. How will I receive CASGEVY? Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It’s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment. After receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment. STEP 1: Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week. During this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY. STEP 2: After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider. STEP 3: Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY. STEP 4: One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time. After the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home. What should I avoid after receiving CASGEVY? Do not donate blood, organs, tissues, or cells at any time in the future What are the possible or reasonably likely side effects of CASGEVY? The most common side effects of CASGEVY include: Low levels of platelet cells, which may reduce the ability of blood to clot and may cause bleeding Low levels of white blood cells, which may make you more susceptible to infection Your healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms: fever chills infections severe headache abnormal bruising prolonged bleeding bleeding without injury such as nosebleeds; bleeding from gums; blood in your urine, stool, or vomit; or coughing up blood These are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. General information about the safe and effective use of CASGEVY Talk to your healthcare provider about any health concerns. Please see full Prescribing Information including Patient Information for CASGEVY. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, YouTube and Twitter/X. (VRTX-GEN) Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Haydar Frangoul, M.D., M.S., and Franco Locatelli, M.D., Ph.D., in this press release, and statements regarding our expectations for and the anticipated benefits of CASGEVY, our plans to share longer-term data for CASGEVY from the CLIMB clinical trials and to share health economics abstracts at the EHA Congress, and our plans for and design of the CLIMB studies. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at . You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. View source version on businesswire.com: Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 Source: Vertex Pharmaceuticals Incorporated View this news release online at:

Clinical ResultCell TherapyDrug ApprovalClinical Study

15 Apr 2024

·www.prnewswire.com

Emmaus Life Sciences Reports Delayed Filing of Annual Report

TORRANCE, Calif., April 15, 2024 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTCQX: EMMA), a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, reported today that the filing of its Annual Report on Form 10-K for the year ended December 31, 2023 will be delayed. "We had hoped to file our Annual Report by today's filing deadline but are still working with our independent registered public accounting firm to complete its review of our accounting for the disposition of our equity interest in a variable interest entity, or VIE, near the end of 2023. We anticipate filing the Form 10-K within the next week or so," explained Willis Lee, Chairman of the Board and Co-president of Emmaus. About Emmaus Life Sciences Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease. Endari® (L-glutamine oral powder), indicated to reduce the acute complications of sickle cell disease in adults and children 5 years and older, is approved for marketing in the United States, Israel, Kuwait, Qatar, the United Arab Emirates, Bahrain and Oman and is available on a named patient or early access basis in France, the Netherlands, and the Kingdom of Saudi Arabia, where Emmaus' application for marketing authorization is awaiting final action by the Saudi Food & Drug Authority. For more information, please visit . About Endari® (prescription grade L-glutamine oral powder) Endari®, Emmaus' prescription grade L-glutamine oral powder, was approved by the U.S. Food and Drug Administration (FDA) in July 2017 for treating sickle cell disease in adult and pediatric patients five years of age and older. Indication Endari® is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients five years of age and older. Important Safety Information The most common adverse reactions (incidence >10 percent) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremities, back pain, and chest pain. Adverse reactions leading to treatment discontinuation included one case each of hypersplenism, abdominal pain, dyspepsia, burning sensation, and hot flash. The safety and efficacy of Endari® in pediatric patients with sickle cell disease younger than five years of age has not been established. For more information, please see full Prescribing Information of Endari® at: . About Sickle Cell Disease There are approximately 100,000 people living with sickle cell disease (SCD) in the United States and millions more globally. The sickle gene is found in every ethnic group, not just among those of African descent; and in the United States an estimated 1-in-365 African Americans and 1-in-16,300 Hispanic Americans are born with SCD.1 The genetic mutation responsible for SCD causes an individual's red blood cells to distort into a "C" or a sickle shape, reducing their ability to transport oxygen throughout the body. These sickled red blood cells break down rapidly, become very sticky, and develop a propensity to clump together, which causes them to become stuck and cause damage within blood vessels. The result is reduced blood flow to distal organs, which leads to physical symptoms of incapacitating pain, tissue and organ damage, and early death.2 1Source: Data & Statistics on Sickle Cell Disease – National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, December 2020. 2Source: Committee on Addressing Sickle Cell Disease – A Strategic Plan and Blueprint for Action -- National Academy of Sciences Press, 2020. Forward-looking Statements This press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding the anticipated filing of the company's Annual Report on Form 10-K for the year ended December 31, 2023, and actual results may differ materially. Such forward-looking statements speak only as of the date they are made, and Emmaus assumes no duty to update them, except as may be required by law. Company Contact: Emmaus Life Sciences, Inc. Willis Lee Chairman and Co-president (310) 214-0065, Ext. 1130 [email protected] SOURCE Emmaus Life Sciences, Inc.

Drug Approval

02 Feb 2024

·www.prnewswire.com

Emmaus Life Sciences Receives Marketing Authorization for Puerto Rico

TORRANCE, Calif., Feb. 2, 2024 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTCQX: EMMA), a leader in sickle cell disease (SCD) treatment, today announced that it has received marketing authorization from the Puerto Rico Department of Health for Endari® (L-glutamine oral powder). This approval marks a significant milestone in Emmaus' mission to improve the lives of people with SCD around the world and provides access to this important therapy for the patients living with SCD in Puerto Rico. In addition, Endari® is approved in the United States, Israel, United Arab Emirates, Kuwait, Qatar, Bahrain, and Oman. "We are thrilled to bring Endari® to Puerto Rico, where the SCD community has faced significant challenges in accessing innovative treatments," said George Sekulich, Co-President of Emmaus Life Sciences. "This approval is a testament to our commitment to working with regulatory authorities and patient advocacy groups to expand access to Endari® for all who need it." Endari® is the first FDA-approved oral glutamine therapy for the reduction of acute complications of SCD in adult and pediatric patients five years and older. Clinical studies have shown that Endari® can significantly reduce the frequency of pain crises, hospitalizations, and other acute complications of SCD. Emmaus is committed to working with healthcare providers and payers in Puerto Rico to ensure that Endari® is accessible to all eligible patients. The company is also working to raise awareness of SCD and the benefits of Endari® through educational programs for healthcare providers and patients. About Emmaus Life Sciences Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company engaged in the discovery, development, marketing and sale of innovative treatments and therapies, including those in the rare and orphan disease categories. For more information, please visit . About Endari® (prescription grade L-glutamine oral powder) Indication - Endari® is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients five years of age and older. Important Safety Information The most common adverse reactions (incidence >10 percent) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremities, back pain, and chest pain. Adverse reactions leading to treatment discontinuation included one case each of hypersplenism, abdominal pain, dyspepsia, burning sensation, and hot flash. The safety and efficacy of Endari® in pediatric patients with sickle cell disease younger than five years of age has not been established. For more information, please see full Prescribing Information of Endari® at: . About Sickle Cell Disease There are approximately 100,000 people living with sickle cell disease (SCD) in the United States and millions more globally. The sickle gene is found in every ethnic group, not just among those of African descent; and in the United States an estimated 1-in-365 African Americans and 1-in-16,300 Hispanic Americans are born with SCD.1 The genetic mutation responsible for SCD causes an individual's red blood cells to distort into a "C" or a sickle shape, reducing their ability to transport oxygen throughout the body. These sickled red blood cells break down rapidly, become very sticky, and develop a propensity to clump together, which causes them to become stuck and cause damage within blood vessels. The result is reduced blood flow to distal organs, which leads to physical symptoms of incapacitating pain, tissue and organ damage, and early death.2 1Source: Data & Statistics on Sickle Cell Disease – National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, December 2020. 2Source: Committee on Addressing Sickle Cell Disease – A Strategic Plan and Blueprint for Action -- National Academy of Sciences Press, 2020. Forward-looking Statements This press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements are subject to numerous assumptions, risks and uncertainties which change over time, including the risk that the authorization does not lead to significant sales of Endari® in Puerto Rico and uncertainties related to Emmaus' working capital and ability to carry on its existing operations and obtain needed financing and other factors previously disclosed in the company's reports filed with the Securities and Exchange Commission, and actual results may differ materially. Such forward-looking statements speak only as of the date they are made, and Emmaus assumes no duty to update them, except as may be required by law. Company Contact: Emmaus Life Sciences, Inc. Willis Lee Co-President and Chief Operating Officer (310) 214-0065, Ext. 1130 [email protected] SOURCE Emmaus Life Sciences, Inc.

Drug Approval

100 Deals associated with L-Glutamine

External Link

KEGG	Wiki	ATC	Drug Bank
D00015	L-Glutamine	A16AA03	DB00130

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Anemia, Sickle Cell	US	Emmaus Medical, Inc.	07 Jul 2017
Short Bowel Syndrome	US	Emmaus Medical, Inc.	10 Jun 2004

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Beta-Thalassemia	Phase 3	US	Emmaus Medical, Inc.	01 May 2010
Gastrointestinal Neoplasms	Phase 3	FR	Nestlé SA	01 Oct 2007
Diverticulitis	Phase 2	US	Emmaus Medical, Inc.	31 Oct 2021
Advanced Pancreatic Adenocarcinoma	Phase 1	US	Emmaus Medical, Inc.	13 May 2021
Pancreatic Ductal Adenocarcinoma	Phase 1	US	Emmaus Medical, Inc.	13 May 2021
Pancreatic Cancer	Phase 1	US	Emmaus Medical, Inc.	01 Apr 2021
Diverticulosis, Colonic	Phase 1	US	Emmaus Medical, Inc.	19 Jul 2019
Diverticulosis, Esophageal	Phase 1	US	Emmaus Medical, Inc.	19 Jul 2019
Diabetes Mellitus, Type 2	Phase 1	US	Emmaus Medical, Inc.	24 Jun 2019
Sepsis	Preclinical	CN	Third Military Medical University	20 Dec 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Pubmed	Not Applicable	MELAS Syndrome	9	High-dose oral glutamine supplementation	kakiqyhdrg(wkonyumygo) = another patient was admitted for an epileptic status without stroke-like episode jivhbvctjc (rwwxlnymnr ) View more	-	05 Nov 2022
NCT01952847 (CTgov)	Phase 3	Neoplasms \| Mucositis \| Esophagitis ... View more	77	Questionnaires+Glutamine (Radiation Therapy to Esophagus Patient Group - Glutamine)	bpeuopkhzw(ylfcqhyvgl): P-Value = 0.73 View more	-	15 Jun 2022
				Questionnaires (Radiation Therapy to Esophagus Patient Group - Placebo)
EHA2022	Phase 3	Anemia, Sickle Cell	19	L-glutamine	wzgexvwrqt(ebdastiyts) = bqbgecncoa lklcwhmknt (xpueuogqwd ) View more	-	12 May 2022
NCT01048905 (CTgov)	Phase 2	Thalassemia \| Associated Pulmonary Arterial Hypertension \| Anemia, Sickle Cell	13	L-Glutamine	zkzamzrlpm(olbnwagfvm) = vnuiaaoulz ujnuxeigmc (gklwonbhjk, wabcwrozgl - onnelyhyxx) View more	-	10 Jun 2021
NCT00586209 (CTgov)	Phase 2	Anemia, Sickle Cell \| Thalassemia	15	L-Glutamine (L-glutamine)	fstdvbgduv(tqaahtyjkc) = hfqciqwwdk rkoonowvgh (lyrvlozzls, lqkoqgylqt - tdzjqufzbv) View more	-	02 Feb 2021
				Placebo (Placebo)	fstdvbgduv(tqaahtyjkc) = wceyojukmn rkoonowvgh (lyrvlozzls, xfompbzrch - xjqswgrlwg) View more
NCT00125788 (CTgov)	Phase 2	Thalassemia \| Anemia, Sickle Cell	70	L-glutamine (Investigational Product)	ssjpuvhjsz(rtwifwarbg) = xamzlgseiz nrviwttktd (cguwglijpe, pimfitpfmk - apquuqhjvs) View more	-	29 Jan 2021
				Placebo (Placebo)	ssjpuvhjsz(rtwifwarbg) = choumjiaoo nrviwttktd (cguwglijpe, rtgpnhkyal - thbyktmcsa) View more
NCT01534663 (GLUTFISH-HF, CTgov)	Phase 2/3	Hydrops Fetalis \| Heart Failure	31	Placebo (Placebo)	ahdgshhnnw(bljiezhhol) = cneulysvps winlzrlnob (sriucyczyb, jfnqmnznfc - nlbnaqodaq) View more	-	22 Dec 2020
				Glutamine (Glutamine/Fishoil)	ahdgshhnnw(bljiezhhol) = ssubvnmyct winlzrlnob (sriucyczyb, ktncjvusdv - cncdrjpqac) View more
NCT01983969 (CTgov)	Phase 1/2	Refractory Lymphoma	61	Azacitidine (Azacitidine Dose Level 1)	jemzkycbvm(imnvhnejdp) = hkvpyjdbud sujdbgqtaz (mnohebkewc, goctlrdmib - azuvwibxib) View more	-	27 Jan 2020
				Azacitidine (Azacitidine Dose Level 2)	jemzkycbvm(imnvhnejdp) = xfbaqwslpf sujdbgqtaz (mnohebkewc, motiinuaul - nacowjvgzj) View more
NCT01783522 (CTgov)	Phase 2	Drug intoxication \| Multiple Myeloma \| Peripheral Nervous System Diseases	9	quality-of-life assessment+glutamine (Arm I (Preventative Nutritional Supplementation))	mhqpfsohjq(sazymrjhkc) = wisqeyavbm vsdtrzpdqj (zskotqvjxa, kelzlabukv - biznlgetti) View more	-	22 Oct 2019
				placebo (Arm II (Placebo))	mhqpfsohjq(sazymrjhkc) = eofzrgchcb vsdtrzpdqj (zskotqvjxa, rwrueefvjf - ympcgswrro) View more
NCT01414244 (Pubmed \| Gut)	Phase 2	Irritable Bowel Syndrome	106	Glutamine (5g/t.i.d.)	gecqtdjflp(mejbupqksf) = czarlyuqlb miqjeqcsok (enbbselagd )	Positive	01 Jun 2019

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