Limb-Girdle Muscular Dystrophy Type 2A

Muscular dystrophies are a group of genetic muscle disorders characterized by progressive muscle weakness. There are more than 40 different variations, and the most common ones in children are Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). LAS VEGAS, Nov. 6, 2023 /PRNewswire/ -- Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either be hereditary or occur as a spontaneous genetic mutation. Additionally, there may be multiple genetic subtypes within each specific type of muscular dystrophy, and individuals with the same subtype may exhibit varying symptoms. DelveInsight has recently released a series of epidemiology-based market reports focusing on muscular dystrophy including Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Limb-girdle Muscular Dystrophy. These reports include a comprehensive understanding of current treatment practices, historical and forecasted patient pool, emerging drugs, market share of individual therapies, and historical and forecasted market size from 2019 to 2032 segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Additionally, the reports feature exhaustive analysis of prominent companies working with their emerging candidates in different stages of clinical development. Let's deep dive into the assessment of these muscular dystrophy reports market individually. Becker Muscular Dystrophy Market Becker muscular dystrophy (BMD) is a genetic disorder that primarily impacts muscle structure. It leads to the gradual deterioration of limb, cardiac, and skeletal muscles. Interestingly, involuntary muscles remain unaffected. BMD is considered a milder form of Duchenne muscular dystrophy (DMD). Typically, BMD manifests in one's teenage years or early adulthood, and its progression is slower and less predictable compared to DMD. As per DelveInsight analysis, it can be observed that prevalence of BMD is high in the 7MM, and similarly, high rate of treatment is seen in patients. The approach to BMD treatment centers on symptom management since there is no definitive cure available. Instead, there are treatments designed to alleviate symptoms and enhance muscle functionality. It's essential for individuals living with BMD to maintain their physical conditioning and muscle usage. While there are no FDA-approved drugs specifically for BMD, researchers are actively investigating various therapeutic interventions. As per DelveInsight analysis, the dynamics of the becker muscular dystrophy market are anticipated to change in the coming years owing to the positive outcomes of the emerging pipeline candidates during the developmental stage by key players that are under phase III, II and I clinical development have the potential to create a significant positive shift in BMD market size. Becker Muscular Dystrophy Pipeline Therapies and Companies EDG-5506: Edgewise Therapeutics, Inc./Medpace, Inc. Vamorolone: ReveraGen BioPharma, Inc./Santhera Pharmaceuticals For a comprehensive view of the Becker muscular dystrophy market, check out the Becker Muscular Dystrophy Market Assessment Myotonic Dystrophy Market Myotonic dystrophy, an umbrella term for two rare genetic disorders with autosomal dominant inheritance patterns, impacts various bodily systems, featuring a clinical triad of progressive muscle weakness, myotonia, and early-onset cataracts. These disorders are classified into two primary types: DM type 1 (DM1) and DM type 2 (DM2). DelveInsight estimated that the total diagnosed prevalent patient population of myotonic dystrophy in the 7MM countries was close to 80K cases in 2021. As per the estimates, the US had the highest patient population of myotonic dystrophy in 2021. Although numerous pre-clinical advancements have been made, there is currently no targeted disease-modifying treatment available. The mainstay of management primarily involves monitoring for complications and adhering to standard care practices, such as utilizing assistive devices, administering hormone therapy, and providing pain relief. Several clinical studies have systematically assessed the efficacy of therapeutic agents in managing the condition. However, the scarcity of scientific evidence, coupled with the complex and diverse manifestations of the disease, poses significant challenges for healthcare providers when it comes to identifying and selecting appropriate medications. To tap the uncovered myotonic dystrophy market, few players have targeted the unmet need fortreatment. As per DelveInsight analysis, the myotonic dystrophy market is expected to grow from USD 80 million in 2021 at a significant CAGR by 2032. The current pipeline consists of both mid and late-stage candidates. The few promising therapies inthe mid and late-stage development are expected to experience significant reconstitution during the forecastperiod. Hence, more research and studies are needed to develop potential therapies that cater to the unmet need for curative and disease-modifying therapy in myotonic dystrophy patients. Myotonic Dystrophy Pipeline Therapies and Companies AMO-02 (tideglusib): AMO Pharma Limited Mexiletine: Lupin Ltd. Pitolisant: Harmony Biosciences, LLC AOC 1001: Avidity Biosciences, Inc. DYNE-101: Dyne Therapeutics To gain a deeper understanding of the myotonic dystrophy market, be sure to explore the Myotonic Dystrophya Market Outlook Facioscapulohumeral Muscular Dystrophy (FSHD) Market Facioscapulohumeral muscular dystrophy (FSHD), a genetically inherited disorder, progressively weakens muscles and significantly reduces the functional capacity of those affected. Recent years have witnessed substantial progress in our understanding of how epigenetic factors play a role in this condition. FSHD can be categorized into two subtypes based on the mechanisms of reduced methylation and failed epigenetic repression. Type 1 accounts for approximately 95% of FSHD cases and is characterized by the loss of D4Z4 repeats. The remaining 5% fall into Type 2, where methylation and epigenetic changes occur independently of deletions. Regardless of the subtype, FSHD leads to the same outcome. As per DelveInsight analysis, in the year 2021, the total prevalent cases of FSHD were 78K in the 7MM which are expected to grow during the study period, i.e., 2019–2032. As of now, there are no officially approved therapies for managing FSHD. However, individuals dealing with FSHD can find relief by utilizing orthotic devices designed to support their feet. These devices are particularly effective in addressing common FSHD symptoms like foot drop and shoulder weakness. Depending on the degree of foot drop, a custom-made ankle-foot orthosis (AFO) can provide significant benefits. In cases where knee extensor weakness may worsen gait with an AFO, alternatives like floor reaction ankle-foot orthosis (FRAFO) or advanced knee-ankle-foot orthosis (KAFO) may be considered. Regular exercise, especially hydrotherapy, plays a crucial role in preserving mobility and pain management. Additionally, weight management, potentially through dietary adjustments, is essential to reduce strain on already weakened muscles. It's important to note that there is currently no cure for FSHD, but there are treatments and devices available to alleviate many of its symptoms. Non-steroidal anti-inflammatory drugs (NSAIDs) are commonly prescribed to enhance comfort and mobility. According to DelveInsight, the FSHD market in 7MM is expected to witness a major change in the study period (2019–2032). As per estimates, in 2021, the total FSHD market size was USD 25.55 million which is expected to rise by 2032. This increase in market size is mainly due to extensive research and development activities of pharmaceutical companies, along with the expected launch of approved therapies. Facioscapulohumeral Muscular Dystrophy Pipeline Therapies and Companies Losmapimod: Fulcrum Therapeutics/GSK GYM329 (RO7204239/RG 6237): Roche Explore in-depth for a comprehensive understanding of the FSHD Clinical Trials Limb-girdle Muscular Dystrophy Market Limb-girdle muscular dystrophies (LGMD), also known as pelvofemoral muscular dystrophy or proximal muscular dystrophy, encompass a group of infrequent genetic disorders characterized by the progressive wasting and weakening of voluntary muscles located in the hip and shoulder regions. This muscle weakness and atrophy gradually advance and can eventually impact other muscle groups in the body. As per data from the National Organization for Rare Disorders, LGMD affects both males and females in roughly equal proportions. The exact prevalence of LGMD remains uncertain, but estimates suggest a range between one in 14,500 to one in 123,000 individuals. It's important to note that the age at which symptoms manifest can vary significantly, even within the same family. Additionally, the relative frequencies of the different LGMD types differ across populations, though it is worth mentioning that LGMD2G, 2H, and 2J are exceedingly rare on a global scale. Currently, there is no known cure for LGMD. However, there are supportive therapies designed to manage and alleviate its symptoms. The approach to LGMD management is individualized, focusing on addressing specific symptoms that each patient experiences. Treatment options may involve physical and occupational therapy to enhance muscle strength and prevent contractures, the utilization of assistive devices like canes, braces, walkers, or wheelchairs to aid mobility, corrective surgery for skeletal issues like scoliosis, and regular monitoring of cardiac and respiratory health for potential complications associated with certain LGMD subtypes. According to DelveInsight's' estimates, the limb girdle muscular dystrophy market in 7MM is expected to show a positive growth, during the forecast period (2023–2032), mainly attributed to anticipated launch of emerging therapy and the increasing cases of LGMD. Limb-girdle Muscular Dystrophy Pipeline Therapies and Companies SRP-6004: Sarepta Therapeutics, Inc. LION-101: Asklepios Biopharmaceutical, Inc. BBP-418 (ribitol): ML Bio Solutions, Inc. To access a complete analysis of the limb-girdle muscular dystrophy market, visit Limb-girdle Muscular Dystrophy Market Assessment Trending Genetic Disorders Reports Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DMD companies, including Pfizer, Santhera Pharmaceuticals, ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Sarepta Therapeutics, Capricor Therapeutics, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, among others. Alpha-1 Antitrypsin Deficiency Market Alpha-1 Antitrypsin Deficiency Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key A1ATD companies, including Arrow Head Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, among others. Wilson Disease Market Wilson Disease Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wilson disease companies, including Orphalan, AstraZeneca, Vivet Therapeutics, Pfizer, Ultragenyx Pharmaceutical, among others. Cystic Fibrosis Market Cystic Fibrosis Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key cystic fibrosis companies, including Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., Vertex Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . 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WESTPORT, Conn., Sept. 21, 2023 /PRNewswire/ -- Today, Coalition to Cure Calpain 3 (C3) in partnership with the National Organization for Rare Disorders (NORD®) launched a study with global reach to research Calpainopathy (including limb-girdle muscular dystrophy type 2A/LGMDR1 and limb-girdle muscular dystrophy type 1I/LGMDD4), a rare form of muscular dystrophy that causes progressive muscle weakness and wasting. Calpainopathy currently has no cure. Continue Reading Coalition to Cure Calpain 3 in Partnership with the National Organization for Rare Disorders (NORD®) Launches LGMD2A/Calpainopathy Registry The new study, the LGMD2A/Calpainopathy Registry, creates a platform to bring the Calpainopathy community together and collect patient data that is an essential requirement for policy makers, academic researchers, and therapeutics companies to advance treatments for this disease. Additionally, the study will help identify individuals with Calpainopathy who might be willing to take part in other research studies or clinical trials. The LGMD2A/Calpainopathy Registry replaces C3's original patient registry. While the original registry served primarily to contact Calpainopathy patients, the new registry will have the capability to collect longitudinal data by having participants periodically update their information. This information is vital to help us understand the progression of symptoms over time, which will inform patient care, drug development, and clinical trial design. "Our goal is to enroll as many patients, or their parents or legal guardians, as possible," notes Jordan Boslego, C3 President. "The success of the registry is dependent upon community participation, and it's essential that the 1000+ patients in our current Global LGMD2A Registry take the time to register on this new platform." "The LGMD2A/Calpainopathy Registry will provide a complete picture of each patient's experience with this disease," shares Dr. Jennifer Levy, C3 Scientific Director. "We are launching this initiative to help fill in the missing information researchers and medical experts need to advance research and one day, find a cure." "This new study has tremendous promise as a strong partnership that engages the patient community and addresses current knowledge gaps for Calpainopathy. NORD is thrilled to be a part of driving research and innovation-based outcomes for all the families in the Calpainopathy community," says Aliza Fink, Director of Research Programs, NORD. For more information, visit . About Coalition to Cure Calpain 3 Coalition to Cure Calpain 3 (C3), a U.S.-based, public, 501(c)(3) non-profit organization, was founded in 2010 to fulfill a mission to drive high-quality research while educating the global community about the muscle-wasting disease limb-girdle muscular dystrophy type 2A (LGMD2A), a form of calpainopathy. C3 has committed over $2 million in grants to international leaders in the muscular dystrophy field to support the development of essential research tools, the identification of outcome assessments that will be used in future clinical trials, and the testing of potential therapies in animal models to determine if they can be safe and effective for LGMD2A. For more information on C3's efforts to treat and cure this disease, visit . About National Organization for Rare Disorders, Inc. (NORD®) The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit . rarediseases.org. Media Contacts Jennifer Levy, C3 [email protected] (203)429-4383 Marketing, NORD [email protected] SOURCE Coalition to Cure Calpain 3

Earlier this year, Lululemon’s founder Chip Wilson went public with his rare disease diagnosis of facioscapulohumeral muscular dystrophy type 2, or FSHD type 2, in which the muscles degenerate over time. Wilson also committed $100 million to find a cure for FSHD through a new venture, Solve FSHD. Chip Wilson Now, up to $10 million of that commitment is going to Vita Therapeutics, a Baltimore-based cell therapy biotech, and Vita will be adding a discovery-phase FSHD cell therapy to its pipeline. Solve FSHD’s executive director Eva Chin will also be joining Vita’s board. The money from Solve FSHD is part of Vita’s $31 million Series B. Cambrian BioPharma, which launched Vita, also led this round of funding, followed by Riptide Ventures, Cedars Sinai, TEDCO, and others. Last year, Cambrian, the anti-aging portfolio company headed by atai co-founder Christian Angermayer, unveiled Vita with a $32 million Series A. UPDATED: Can a cell therapy treat muscular dystrophy? A German billionaire's anti-aging startup is trying to find out Most of the new cash infusion will go toward Vita’s lead program, dubbed VTA-100. The candidate is a cell therapy for limb-girdle muscular dystrophy type 2a (LGMD2A), the most common type of LGMD. While it’s still in preclinical studies, Vita’s CEO Doug Falk said the company hopes to reach the clinic within 18 months. “We’re really on the verge of kicking off the IND-enabling studies. In parallel to those studies, we’re going to be doing the GMP manufacturing that’s needed to go ahead and file an IND,” Falk told Endpoints News . And aside from its muscular dystrophy programs, Vita has an immunotherapy candidate, VTA-300, that it hopes to bring up from discovery to the preclinical stage as well, Falk noted. And it will be looking to add 10-15 employees to its current headcount of around 30. Sarepta is developing a number of gene therapies for LGMD, though its candidate for type 2a specifically is likewise in preclinical studies. But its LGMD candidate SRP-9003 in clinical studies has shown promising early results so far. Sarepta reveals positive results in limb-girdle gene therapy trial. What does that mean for DMD? On the FSHD front, previous attempts to treat the disease have seen little success. In 2019, Acceleron scrapped its program after patients didn’t have functional improvements on the drug. And Fulcrum’s attempt with a drug targeting DUX4, licensed from GSK, failed a Phase II study — though the biotech is pushing onward with Phase III. But aside from Vita, there are a handful of early candidates. Just last week, Sanofi licensed an antibody-RNA conjugate for FSHD from miRecule, an NIH vet-led startup which also got $1 million from Solve FSHD. And Dyne Therapeutics has its own DUX4-targeted preclinical candidate.