A Phase 2a, Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients with Hutchinson-Gilford Progeria Syndrome (HGPS)

Researchers will compare treatment with progerinin plus lonafarnib vs lonafarnib alone to assess optimal dosing, safety, tolerability, and pharmacokinetics in patients with Hutchinson-Gilford Progeria Syndrome (HGPS). Subjects in the randomized study arms will continue to take the standard of care (SOC), lonafarnib, and will be randomized to either take SOC alone or in combination with progerinin.

Start Date01 Jan 2025

Sponsor / Collaborator

PRG S&T Co., Ltd

NCT05953545

/ WithdrawnPhase 2IIT

Open Label Phase 2 Clinical Study of Peginterferon Lambda and Lonafarnib Boosted With Ritonavir 48-Week Combination Therapy for Delta Hepatitis

Background:
Chronic hepatitis D is a serious liver disease caused by a virus. Currently, no medications are approved to treat chronic hepatitis D.
Objective:
To test a combination of 3 drugs in people with chronic hepatitis D.
Eligibility:
People 18 years or older with chronic hepatitis D.
Design:
Participants will be in the study about 2 years. They will have 3 inpatient stays of 3 to 5 days.
Participants will be screened. They will have a physical exam with blood tests. They will have a test of their heart function and an ultrasound: a wand that uses sound waves to create images of the liver will be rubbed over the skin on their torso.
Participants will stay in the clinic for a 3-day baseline visit. They will have imaging scans, an eye exam, and a visit with a reproductive specialist. They will have a liver biopsy: about 1 inch of liver tissue will be removed, either with a tube inserted through a vein in the neck, or with a needle inserted through the participant s side.
Participants will take the study drugs for 48 weeks. Two of them are tablets taken twice a day at home; 1 is a shot administered once a week. Participants will begin taking the drugs during a 5-day stay in the clinic. Then they will have 15 outpatient visits while taking the drugs and 7 more after they finish.
The last 3-day clinic stay will be 6 months after participants finish taking the drugs. The liver biopsy, imaging scans, and other tests will be repeated.

Start Date22 May 2024

Sponsor / Collaborator

National Institute of Diabetes & Digestive & Kidney Diseases

NCT05229991

/ Active, not recruitingPhase 3IIT

Once Daily (QD) Dosing of Lonafarnib (LNF) Co-administered With Ritonavir (RTV) for Treatment of Chronic Hepatitis D Virus Infection

Open label, single arm, multi-center clinical trial of lonafarnib 50 mg QD plus ritonavir 200 mg QD, administered orally, over a 48-week treatment period, with a 24-week post-treatment follow-up period, in patients with chronic Hepatitis D Virusinfection.
Objectives: To evaluate the safety and tolerability of once daily dosing of lonafarnib 50 mg with ritonavir 200 mg over a 48-week treatment period.
To evaluate the effect of once daily dosing of lonafarnib 50 mg with ritonavir 200 mg over a 48-week treatment period with a 24-week post-treatment follow-up on HDV viral levels.
Trial population: Up to 30 patients with chronic HDV infection with detectable HDV RNA and compensated liver disease.

Start Date15 May 2021

Sponsor / Collaborator

Soroka University Medical Center

[+1]

100 Clinical Results associated with Lonafarnib

100 Translational Medicine associated with Lonafarnib

100 Patents (Medical) associated with Lonafarnib

440

Literatures (Medical) associated with Lonafarnib

01 Feb 2025·Journal of Cachexia Sarcopenia and Muscle

Lonafarnib Protects Against Muscle Atrophy Induced by Dexamethasone

Article

Author: Mai, Van‐Hieu ; Dong, Dalong ; Han, Kyudong ; Hyun, Jung Keun ; Park, Sunghyouk ; Mun, Seyoung ; Bae, Sanghoon

ABSTRACT:

Background:

Muscle atrophy, including glucocorticoid‐induced muscle wasting from treatments such as dexamethasone (DEX), results in significant reductions in muscle mass, strength and function. This study investigates the potential of lonafarnib, a farnesyltransferase inhibitor, to counteract DEX‐induced muscle atrophy by targeting key signalling pathways.

Methods:

We utilized in vitro models with C2C12 myotubes treated with DEX and in vivo models with Caenorhabditis elegans and DEX‐treated Sprague–Dawley rats. Myotube morphology was assessed by measuring area, fusion index and diameter. Muscle function was evaluated by grip strength and compound muscle action potential (CMAP) in the gastrocnemius (GC) and tibialis anterior (TA) muscles. Molecular mechanisms were explored through RNA sequencing and Western blotting to assess changes in mitochondrial function and muscle signalling pathways.

Results:

Lonafarnib (2 μM) significantly improved myotube area (1.49 ± 0.14 × 105 μm2 vs. 1.03 ± 0.49 × 105 μm2 in DEX, p < 0.05), fusion index (18.73 ± 1.23% vs. 13.3 ± 1.56% in DEX, p < 0.05) and myotube diameter (31.89 ± 0.89 μm vs. 21.56 ± 1.01 μm in DEX, p < 0.05) in C2C12 myotubes. In C. elegans, lonafarnib (100 μM) increased the pharyngeal pumping rate from 212 ± 7.21 contractions/min in controls to 308 ± 17.09 contractions/min at day 4 (p < 0.05), indicating enhanced neuromuscular function. In DEX‐induced atrophic rats, lonafarnib improved maximal grip strength (DEX: 13.91 ± 0.78 N vs. 1 μM lonafarnib: 16.18 ± 0.84 N and 5 μM lonafarnib: 16.71 ± 0.83 N, p < 0.05), increased muscle weight in GC, and enhanced CMAP amplitudes in both GC and TA muscles. Western blot analysis showed that lonafarnib treatment upregulated UCP3 and ANGPTL4 and increased phosphorylation of mTOR and S6 ribosomal protein (p < 0.05), indicating enhanced mitochondrial function and protein synthesis. Knockdown models further demonstrated that lonafarnib could partially rescue muscle atrophy phenotypes, indicating its action through multiple molecular pathways.

Conclusions:

Lonafarnib mitigates dexamethasone‐induced muscle atrophy by enhancing mitochondrial function and activating anabolic pathways. These findings support further investigation of lonafarnib as a therapeutic agent for muscle atrophy in clinical settings.

09 Jan 2025·JCI Insight

Repurposing of lonafarnib as a treatment for SARS-CoV-2 infection

Article

Author: Jiang, Jian-Kang ; Jang, Eun Sun ; Zheng, Wei ; Coutermarsh-Ott, Sheryl ; Maio, Nunziata ; Hu, Zongyi ; Ricardo-Lax, Inna ; Li, Bing ; Khan, Mohsin ; Leek, Madeleine ; Ivester, Hannah M ; Grieshaber, Ailis ; Allen, Irving C ; Liang, T Jake ; Park, Seung Bum ; Ye, Yihong ; Huang, Wenwei ; Wang, Amy Q ; Rouault, Tracey ; Xu, Xin ; Irvin, Parker ; Zhang, Qi ; Rice, Charles

Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), which causes coronavirus disease 2019 (COVID-19), has emerged as a global pandemic pathogen with high mortality. While treatments have been developed to reduce morbidity and mortality of COVID-19, more antivirals with broad-spectrum activities are still needed. Here, we identified lonafarnib (LNF), a Food and Drug Administration-approved inhibitor of cellular farnesyltransferase (FTase), as an effective anti-SARS-CoV-2 agent. LNF inhibited SARS-CoV-2 infection and acted synergistically with known anti-SARS antivirals. LNF was equally active against diverse SARS-CoV-2 variants. Mechanistic studies suggested that LNF targeted multiple steps of the viral life cycle. Using other structurally diverse FTase inhibitors and a LNF-resistant FTase mutant, we demonstrated a key role of FTase in the SARS-CoV-2 life cycle. To demonstrate in vivo efficacy, we infected SARS-CoV-2-susceptible humanized mice expressing human angiotensin-converting enzyme 2 (ACE2) and treated them with LNF. LNF at a clinically relevant dose suppressed the viral titer in the respiratory tract and improved pulmonary pathology and clinical parameters. Our study demonstrated that LNF, an approved oral drug with excellent human safety data, is a promising antiviral against SARS-CoV-2 that warrants further clinical assessment for treatment of COVID-19 and potentially other viral infections.

01 Jan 2025·INTERNATIONAL JOURNAL OF CANCER

RETRACTION: A Multiplicity of Anti‐Invasive Effects of Farnesyl Transferase Inhibitor SCH66336 in Human Head and Neck Cancer

RETRACTION: S. H.Oh, J.‐H.Kang, J. K.Woo, O.‐H.Lee, E. S.Kim, and H.‐Y.Lee, “,” International Journal of Cancer131, no. (2012): 537–547, https://doi-org.libproxy1.nus.edu.sg/10.1002/ijc.26373.The above article, published online on 16 August 2011 in Wiley Online Library (wileyonlinelibrary.com), has been retracted by agreement between the journal Editor‐in‐Chief, Prof. Christoph Plass; the Union for International Cancer Control; and John Wiley & Sons Ltd.Following publication, concerns were raised by third parties regarding Figure 2D. Additional concerns were found during an investigation by the editorial office regarding Figures 1A and 4A. The authors were unable to provide comprehensive original data. The retraction has been agreed because parts of the figures and the underlying data were duplicated. The editors no longer have confidence in the data reported in this article or in the interpretation of the results presented.The authors S. H. O, J.‐H. K., and H.‐Y. L. were informed of the decision to retract; J. K. W., O.‐H. L., and E. S. K. could not be contacted.

News (Medical) associated with Lonafarnib

05 Mar 2025

·www.fiercebiotech.com

Former NIH director Francis Collins retires, calls agency the \'main piston\' in biomedical discovery

Collins first joined the NIH in 1993 to lead the Human Genome Project.\n After a 32-year career at the National Institutes of Health, Francis Collins, M.D., Ph.D., retired from the agency, effective Feb. 28.The NIH “has been rightfully called the ‘crown jewel’ of the federal government for decades,” Collins said in a March 1 statement (PDF). “It has been the greatest honor of my life to be part of this institution in various roles over the last four decades.”Collins served as NIH director from 2009 to 2021, spanning three presidents: Barack Obama, Donald Trump and Joe Biden. After stepping down as director, he briefly served as Biden’s science advisor in 2022. He then returned to the NIH’s National Human Genome Research Institute, where he had served as director from 1993 to 2008. There, Francis led the Human Genome Project, which successfully finished compiling a full human genome in 2003. Throughout his NIH career, Collins’ research group focused on type 2 diabetes and a rare disease called progeria, which causes premature aging. His group’s work helped lead to the first approved therapy for the disease, Sentynl Therapeutics’ Zokinvy (lonafarnib). Sentynl acquired the rights to Zokinvy from original developer Eiger BioPharmaceuticals in 2024.In his statement, Collins hailed the NIH as the “main piston of a biomedical discovery engine that is the envy of the globe,” adding that the agency’s employees “deserve the utmost respect and support of all Americans.”Collins is the latest federal health agency leader to depart since President Donald Trump was reelected and inaugurated for a second term. For example, the NIH’s Deputy Director for Extramural Research Michael Lauer, M.D., left the agency a few weeks ago, while Principal Deputy Director Larry Tabak, Ph.D., retired.Under the new administration, scores of federal employees have been fired, research grants have been canceled and the Elon Musk-led Department of Government Efficiency has terminated leases for 30 FDA facilities across 23 states.

Executive ChangeDrug Approval

02 Oct 2024

·www.biospace.com

US FDA Authorizes Launch of Clinical Trial to Support New Treatment Development for Progeria

New Trial to Study Progerinin Treatment for Ultra-Rare Condition BUSAN, KOREA; PEABODY, MA; and GERMANTOWN, MD / ACCESSWIRE / October 2, 2024 / The United States Food and Drug Administration (FDA) has authorized the start of patient enrollment for a Phase 2a clinical trial of Progerinin, an investigational drug being developed for the treatment of Progeria. Advancing the development of Progerinin could potentially provide an additional treatment for this fatal disease. PRG Science & Technology logo Hutchinson-Gilford Progeria Syndrome (“Progeria”) is an ultra-rare, genetic, “rapid-aging” condition affecting approximately 1 in 18 million people. Without treatment, children with Progeria die of heart disease at an average age of 14.5 years old. Progeria is caused by a random genetic mutation that produces an overabundance of the toxic protein, progerin. The accumulation of progerin occurs with aging in the general population, in the cardiovascular system and elsewhere in the body, such as the skin. In children and young adults with Progeria, the rate of progerin accumulation is highly accelerated, resulting in progressive cellular damage and atherosclerotic heart disease. Progerinin was developed by Korean-based biotech company PRG Science & Technology Co., Ltd. (PRG S&T) with research funding from The Progeria Research Foundation (PRF). The trial is a collaborative effort between PRG S&T, PRF, and trial site Boston Children’s Hospital, and will be managed by contract research organization Amarex. In the Phase 2a randomized open label trial, Progerinin will be administered in combination with the current standard-of-care, Zokinvy, to 10 patients with Progeria and Progeroid Laminopathies (PL) who are one year of age and older. Zokinvy has been shown to increase lifespan in a Progeria mouse model by 25%, and data published in the journal Cells in April 2023 indicated that Progerinin showed a potential increase in Progeria mouse lifespan by 50%. Zokinvy works by blocking the toxic, disease-causing protein, progerin, from developing and attacking normal cells, while Progerinin appears to reduce the level of progerin that accumulates in the body. Currently, Zokinvy is the only treatment for Progeria and PL approved by the FDA (in 2020), the European Medicines Agency (EMA, in 2022) and the Japanese Ministry of Health, Labor and Welfare (MHLW, in 2024). “Since its establishment, PRG S&T has focused on developing rare genetic disease treatments. This clinical trial demonstrates PRG S&T’s R&D capabilities to improve access to new drugs and expand treatment opportunities for children with progeria worldwide,” said Bumjoon Park, president of PRG S&T. “PRG S&T will focus on R&D and technological innovation to accelerate the development of treatments, including preparation for follow-up clinical trials, and lead to the development of new drugs.” “This new trial represents another step forward in our mission to treat and cure these amazing children and young adults with Progeria,” said PRF Executive Director Audrey Gordon, Esq. “Each Progeria clinical trial builds on learnings from our previous trials, so that we are continually making progress toward even better treatments and the cure. Most importantly, we are grateful for the courage and resilience of these children and young adults with Progeria and their families for participating in research programs and contributing to this important mission.” “Patients with Progeria and their families deserve more options,” said Dr. Kush Dhody, President, Amarex Clinical Research. “This is an especially devastating diagnosis given the shortened lifespan it causes. We’re honored to support a study that brings more hope to these patients.” About Progeria Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), and PL are ultra-rare, multisystemic, premature aging diseases that accelerate mortality in young patients. Progeria is caused by a genetic mutation in the LMNA (“lamin A”) gene, and results in a disease-causing abnormal protein called progerin. There are approximately 400-450 children and young adults worldwide with Progeria. Thanks to PRF-funded research, we now know that progerin is produced in all of us as we age, but at a much lower rate than in children with Progeria. Due to this discovery of the biological connection between Progeria, heart disease and aging, finding the cure for one of the rarest diseases on earth could provide keys for treating millions of adults with heart disease and stroke associated with the natural aging process, as well as help the entire aging population. About PRG Science & Technology Co., Ltd. PRG S&T is a research & development company specializing in the treatment of rare genetic diseases. PRG S&T has been developing therapeutics for rare genetic diseases (laminopathies, neurodegenerative diseases, neuro-oncology etc.) with small molecules, which are derived from target sites on aberrant Protein-Protein Interactions (PPI). All four pipelines currently in PRG S&T’s possession have been designated as orphan drugs by the US FDA. Progerinin, the investigational treatment for Progeria has been approved by the FDA to enter phase 2 clinical trials, and is about to enter Phase2 at Boston Children’s Hospital. The non-clinical study of PRG-N-01, the treatment for neurofibromatosis type2, has been completed. IND for Phase I/II of PRG-N-01 has been approved by the Korea FDA and has been administered to patients at Asan Medical Center (South Korea) in June 2024. If these two drugs, Progerinin and PRG-N-01, are developed successfully, they are expected to become first-in-class innovative new drugs. For more information, please visit . About The Progeria Research Foundation The Progeria Research Foundation (PRF) was established in 1999 by the family of Sam Berns, a child with Progeria. Within four years of its founding, the PRF Genetics Consortium discovered the Progeria gene, a collaboration led by Dr. Francis Collins, former Director of the National Institutes of Health (NIH). PRF has funded and co-coordinated all lonafarnib-associated clinical trials for Progeria and Progeroid Laminopathies, conducted at Boston Children’s Hospital, and supports scientists who conduct Progeria research worldwide. PRF’s International Patient Registry includes over 370 children with Progeria in 70 countries. PRF is the only non-profit organization solely dedicated to finding treatments and the cure for Progeria and its aging-related conditions, including heart disease. The organization fills a void, putting these children and Progeria at the forefront of scientific efforts. For more information and to support PRF’s mission, please visit . About Amarex Clinical Research, LLC, an NSF company Amarex Clinical Research, LLC, is a global, full-service Contract Research Organization (CRO) with significant expertise conducting biomedical research. The leadership team’s combined experience includes the design and conduct of several hundred clinical research projects in many therapeutic indications. Amarex provides services in Project Management: Phase I-IV, BE/BA, PK/PD; Regulatory Affairs: FDA Applications and meetings, applications to International Health Authorities, GxP Compliance Audits; Clinical Operations; Adaptive Study Designs; Statistical Analysis; Data Management; Medical Monitoring; Safety and Pharmacovigilance; and General Consulting. Amarex can take your product through the entire approval process, from creating the regulatory approval strategy, to conducting trials, to writing the marketing approval application. Join our growing list of clients with approved products assisted by quality, cost-efficient services. For more information visit . Contact Information Eleanor Maillie PRF emaillie@progeriaresearch.org 978.879.9244 Sunghee Kim PRG S&T shkim@prgst.com Kalli DeWeese Amarex kdeweese@nsf.org Related Images PRG Science & Technology logo Amarex Clinical Research logo PRF logo SOURCE: The Progeria Research Foundation View the original press release on newswire.com.

Phase 2Orphan DrugDrug Approval

06 May 2024

·www.pharmaceutical-technology.com

Sentynl acquires Eiger’s Zokinvy for progeria treatment

The therapy is indicated for treating Hutchinson-Gilford Progeria Syndrome and processing-deficient progeroid laminopathies. Credit: LeviaUA / Shutterstock.com. Zydus Lifesciences subsidiary Sentynl Therapeutics has announced the worldwide acquisition of the Zokinvy (lonafarnib) programme from Eiger BioPharmaceuticals for the treatment of progeria. Zokinvy is the first and only treatment to obtain US FDA approval that targets the cause and symptoms of Hutchinson-Gilford Progeria Syndrome (HGPS) and processing-deficient progeroid laminopathies (PDPL), collectively called progeria. The treatment is indicated for usage in individuals aged 12 months and above. An ultra-rare, fatal genetic condition, progeria causes premature aging in young patients, leading to accelerated mortality. Since its approval in the US in 2020, Zokinvy has also received authorisation in the European Union and Great Britain in 2022, and most recently in Japan in January this year. See Also: Novartis acquires Mariana in $1.75bn deal to strengthen radioligand portfolio Vertex Pharmaceuticals gets grant for treatment of alpha-1 antitrypsin deficiency with compound of formula (i) The latest sale comes after Eiger and its subsidiaries filed for Chapter 11 bankruptcy in the US Bankruptcy Court in April this year. Subsequently, an auction was held as part of Eiger’s court-supervised sale process of Zokinvy. Sentynl emerged as the winning bidder for net base price of $45.2m. The Bankruptcy Court subsequently approved the sale to Sentynl with the transaction finalised on 3 May. Following the acquisition, Sentynl holds the worldwide rights to Zokinvy and will oversee its manufacturing and commercialisation efforts. The new deal will bolster Sentynl’s portfolio of biopharmaceutical products for rare and ultra-rare diseases. Sentynl president and CEO Matt Heck said: “It is an honour to add Zokinvy to our portfolio of products that have a tangible impact on the lives of rare disease patients, whose needs are too often unmet or overlooked. “We are firmly committed to provide best-in-class global access to Zokinvy and are eager to serve the patients and their families affected by progeria. We are grateful to Eiger and The Progeria Research Foundation for their dedicated effort to develop and secure availability of this life-changing product.”

Drug ApprovalAcquisition

100 Deals associated with Lonafarnib

External Link

KEGG	Wiki	ATC	Drug Bank
D04768	Lonafarnib	A16AX	DB06448

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Laminopathies	European Union	Tmc Pharma (Eu Ltd.	18 Jul 2022
Laminopathies	Iceland	Tmc Pharma (Eu Ltd.	18 Jul 2022
Laminopathies	Liechtenstein	Tmc Pharma (Eu Ltd.	18 Jul 2022
Laminopathies	Norway	Tmc Pharma (Eu Ltd.	18 Jul 2022
Hutchinson-Gilford progeria syndrome	United States	Sentynl Therapeutics, Inc.Startup	20 Nov 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Hepatitis D, Chronic	Phase 3	Israel	Eiger BioPharmaceuticals, Inc.	15 May 2021
Hepatitis D, Chronic	Phase 3	New Zealand	Eiger BioPharmaceuticals, Inc.	15 May 2021
Hepatitis D, Chronic	Phase 3	Turkey	Eiger BioPharmaceuticals, Inc.	15 May 2021
Hepatitis D	Phase 3	United States	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Belgium	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Bulgaria	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Canada	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	France	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Germany	Eiger BioPharmaceuticals, Inc.	01 Dec 2018
Hepatitis D	Phase 3	Greece	Eiger BioPharmaceuticals, Inc.	01 Dec 2018

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02527707 (LOWR-4, CTgov)	Phase 2	Hepatitis D, Chronic	15	lonafarnib+Ritonavir	cjljexzidn(iatwadcvki) = eivirkmcib zmcpufaijm (gbbonyznnr, 1.4) View more	-	22 Jun 2023
NCT00425607 \| NCT00916747 (Pubmed \| Genet Med)	Not Applicable	Laminopathies \| Hutchinson-Gilford progeria syndrome	-	Lonafarnib	hqmjgcoskh(czguvookpd) = rtlqtdqqkl eonmfnipkp (viwhbqcsdj )	Positive	12 Dec 2022
NCT02430181 (LOWR-1, CTgov)	Phase 2	Hepatitis D, Chronic	21	lonafarnib+ritonavir	miemtnfwfe(pplessrohl) = hlrlnphlqe zijvvnqtqt (ifgkzqmlpf, myppsxpwgr - vxitnyexyc) View more	-	29 Nov 2022
NCT03600714 (CTgov)	Phase 2	Hepatitis D, Chronic	26	Lonafarnib+Peg-interferon lambda+Ritonavir	pqucpoimyk = tanveysnmu fraxkxrxue (xildsvgyzm, buuzhkadfd - ebcfylsimg) View more	-	01 Dec 2021
LIFT HDV Study (EASL2020)	Phase 2	Hepatitis D HDV RNA \| HBV DNA	26	Peginterferon Lambda	ylkgtugmmi(jynfuderjv) = rjynxqpimn urpsicwxps (aikjjfldas, 2.9–3.8)	-	27 Aug 2020
LIFT HDV Study (EASL2020)	Phase 2	Hepatitis D HDV RNA \| HBV DNA	26	Lonafarnib	ylkgtugmmi(jynfuderjv) = bpgfdruazj urpsicwxps (aikjjfldas, 2.9–4.5)	-	27 Aug 2020
Pubmed Manual	Not Applicable	Hutchinson-Gilford progeria syndrome	258	Lonafarnib	wtbmpoqttz(jppchmegby) = Treatment was associated with a lower mortality rate (hazard ratio, 0.12; 95% CI, 0.01-0.93; P = .04). In the combined cohort, there were 4 deaths (6.3%) among 63 patients in the treated group and 17 deaths (27.0%) among 63 patients in the matched untreated group (hazard ratio, 0.23; 95% CI, 0.06-0.90; P = .04). qsgxnfgfeg (qyqqvzqqvi )	Positive	24 Apr 2018
Pubmed Manual	Not Applicable	Hutchinson-Gilford progeria syndrome	258	control		Positive	24 Apr 2018
NCT00879034 (CTgov)	Phase 2	Laminopathies \| Hutchinson-Gilford progeria syndrome	5	Lonafarnib+Pravastatin+Zoledronic Acid	xetreptreq = kpgowhnorf qmlegmljba (uufqrgmocd, wxhcuzpxxf - qegdwlpqlh) View more	-	31 Jul 2017
NCT00425607 (CTgov)	Phase 2	Laminopathies \| Hutchinson-Gilford progeria syndrome	29	Lonafarnib	eeqkefczfk = imrlwojmuy ipmnznpwjt (yykduojgno, pdpmhsfhcz - qqalyhaebg) View more	-	24 May 2017
NCT01495585 (CTgov)	Phase 2	Hepatitis D, Chronic	14	Placebo (Placebo)	mdopecpaux(zxzmvpnhsf) = quzdieumyv mfghggmrwp (grhyxuutpi, 0.14) View more	-	31 Aug 2016
NCT01495585 (CTgov)	Phase 2	Hepatitis D, Chronic	14	Lonafarnib (Group 1)	mdopecpaux(zxzmvpnhsf) = yizdzehijo mfghggmrwp (grhyxuutpi, 0.54) View more	-	31 Aug 2016

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