Latest news with #FacioscapulohumeralMuscularDystrophy
Malaysian Reserve
17-07-2025
- Health
- Malaysian Reserve
Facioscapulohumeral Muscular Dystrophy Market Gearing Up for Outstanding Expansion Across the 7MM During the Forecast Period (2025-2034)
According to DelveInsight's analysis, the market for facioscapulohumeral muscular dystrophy is anticipated to increase during the forecast period (2025–2034), owing to improved diagnosis, treatment advancements, aging demographics, increased awareness, the launch of emerging therapies, and healthcare spending globally. LAS VEGAS, July 17, 2025 /PRNewswire/ — Facioscapulohumeral muscular dystrophy (FSHD) often starts with muscle weakness in the face, shoulders, and upper arms, and gradually progresses to affect the abdominal muscles, legs, and pelvic area as the disease advances. Symptoms commonly emerge during childhood or adolescence, though they can begin at any stage of life, from infancy to old age. There is considerable variation in symptom onset and severity, even among relatives with the condition. Roughly 80% of individuals carrying the FSHD gene mutation experience symptoms, while the remaining 20% are asymptomatic, either showing no signs or only very mild symptoms that may go unnoticed until later in life. For FSHD1, children of an affected parent have a 50% likelihood of inheriting the disorder. In FSHD2, the inheritance risk depends on the genetic background of both parents, typically ranging from 25% to 50%. In 2024, the United States represented about 45% of all FSHD cases across the 7MM, with numbers projected to rise by 2034. Most US patients in 2024 scored between 7 and 10 on the RICCI scale, indicating moderate to severe levels of muscle dysfunction. Download the report to understand which factors are driving FSHD epidemiology trends @ Facioscapulohumeral Muscular Dystrophy Treatment Algorithm At present, there are no available therapies that can slow, halt, or reverse the progression of muscle weakness in facioscapulohumeral muscular dystrophy. While physical therapy can be beneficial in some cases, it is generally recommended that individuals engage in regular low-resistance and aerobic exercise. Collaborating with a physical therapist is important to create a personalized and safe exercise regimen. Some patients may benefit from surgical stabilization of the scapula to enhance their ability to raise their arms above shoulder level. Scapular fixation involves anchoring the shoulder blades to the ribs and may be performed on one or both sides. This is a complex procedure that should only be undertaken by skilled surgeons after thorough evaluation and discussion with both the surgeon and the neurologist. Additionally, medications such as NSAIDs, opioids, and antidepressants are commonly prescribed to manage FSHD-related pain. Current care practices are centered around managing complications and preserving physical function. Rehabilitation consultations are advised for individuals facing mobility or functional issues. However, there remains a significant unmet need for effective and affordable approved therapies, as existing supportive treatments are often costly and limited in efficacy. Learn more about the FSHD treatment @ New Treatment for Facioscapulohumeral Muscular Dystrophy There remains a significant unmet need in the treatment of FSHD, as current approaches primarily involve off-label therapies. Management strategies are largely aimed at alleviating symptoms and preserving physical function. Although FSHD is a progressive disorder with potentially serious long-term effects, treatment options are still limited to symptomatic care. Several investigational therapies are in development, including GYM329/RO7204239/RG6237 by Roche and Chugai Pharmaceutical, Delpacibart braxlosiran (del-brax) by Avidity Biosciences, EPI-321 by Epicrispr Biotechnologies, among others. Discover which therapies are expected to grab major FSHD market share @ Facioscapulohumeral Muscular Dystrophy Market Report GYM329 (also known as RO7204239 or RG6237) is an experimental anti-myostatin antibody aimed at enhancing skeletal muscle mass and growth. It has been specifically engineered as a 'recycling' and 'sweeping' antibody, meaning it may clear myostatin from the bloodstream more effectively than traditional antibodies. This targeted approach is expected to help address conditions marked by muscle wasting and reduced muscle strength. In November 2022, Roche began a Phase II clinical trial (NCT05548556) in patients with facioscapulohumeral muscular dystrophy, and according to the company, regulatory filing is anticipated after 2028. As per Chugai Pharmaceutical's Q1 2025 update, results from the ongoing MANOEUVRE Phase II study for FSHD are expected in 2025. With proof of concept established, Chugai plans to advance to Phase III trials, set to begin in 2026. Delpacibart braxlosiran (del-brax, formerly AOC 1020) is currently being assessed in the Phase I/II FORTITUDE trial (NCT06547216) in both adult and adolescent participants with FSHD. This study is focused on evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenously administered del-brax. In June 2025, Avidity Biosciences announced that del-brax may qualify for an accelerated approval pathway in the U.S. for FSHD treatment. The company also launched the global Phase III FORWARD trial to serve as a confirmatory study for full regulatory approval. Additionally, Avidity reported positive topline results from the dose-escalation cohorts of the FORTITUDE trial, which will be shared at the 32nd Annual FSHD Society International Research Congress. EPI-321 is an investigational, one-time gene-silencing therapy targeting abnormal DUX4 expression. Administered systemically via a validated AAV vector, EPI-321 has shown strong preclinical results, including effective suppression of DUX4 and preservation of muscle tissue. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA. The company is preparing to launch a global Phase I/II trial in 2025. Discover more about drugs for FSHD in development @ Facioscapulohumeral Muscular Dystrophy Clinical Trials The anticipated launch of these emerging therapies for FSHD are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the FSHD market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for FSHD is expected to grow at a significant CAGR by 2034. This growth is mainly driven by increased awareness, improved diagnostic techniques, and ongoing research into genetic therapies. Rising investment from pharmaceutical companies and the emergence of targeted therapies are expanding treatment options. Regulatory support for orphan diseases is also accelerating drug development. DelveInsight's latest published market report, titled as Facioscapulohumeral Muscular Dystrophy Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the FSHD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The FSHD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of FSHD Total Diagnosed Prevalent Cases of FSHD Type-specific Diagnosed Prevalent Cases of FSHD Gender-specific Diagnosed Prevalent Cases of FSHD Age-specific Diagnosed Prevalent Cases of FSHD Severity-specific Diagnosed Prevalent Cases of FSHD Total Treated Cases of FSHD The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM FSHD market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this FSHD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the FSHD market. Also, stay abreast of the mitigating factors to improve your market position in the FSHD therapeutic space. Related Reports Facioscapulohumeral Muscular Dystrophy Pipeline Facioscapulohumeral Muscular Dystrophy Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key FSHD companies, including Fulcrum Therapeutics, Dyne Therapeutics, Hoffmann-La Roche, aTyr Pharma, Inc., Avidity Biosciences, Inc., among others. Facioscapulohumeral Muscular Dystrophy Epidemiology Forecast Facioscapulohumeral Muscular Dystrophy Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted FSHD epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 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 Sarepta Therapeutics, PTC Therapeutics, Nippon Shinyaku, Santhera Pharmaceuticals, ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Capricor, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, among others. Spinal Muscular Atrophy Market Spinal Muscular Atrophy Market Insights, Epidemiology, and Market Forecast – 2034 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 SMA companies, including Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Therapeutics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact Us Shruti Thakur info@ +14699457679 Logo: View original content:
Yahoo
10-06-2025
- Business
- Yahoo
Avidity Biosciences price target raised to $75 from $65 at Chardan
Chardan analyst Keay Nakae raised the firm's price target on Avidity Biosciences (RNA) to $75 from $65 and keeps a Buy rating on the shares after the company announced topline results from the dose escalation portion of the FORTITUDE trial and that it has received written confirmation from the FDA on an accelerated approval pathway for del-brax in Facioscapulohumeral Muscular Dystrophy using a novel circulating biomarker, cDUX. The firm's new price target reflects an increase in the estimated probability of success to 60% from 50% for del-brax targeting FSHD, the analyst tells investors. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on RNA: Disclaimer & DisclosureReport an Issue Avidity Biosciences Launches Global Phase 3 Study Avidity Biosciences announces results from Phase 1/2 FORTITUDE program Avidity announces accelerated approval regulatory pathway for Del-Brax Avidity Biosciences Reports Q1 2025 Earnings Promising Outlook for Avidity Biosciences: Buy Rating Backed by Strategic Advancements and Product Launch Preparations Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
09-06-2025
- Business
- Yahoo
Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial
Avidity Biosciences, Inc. (NASDAQ:RNA) revealed topline data from the dose escalation cohorts of the delpacibart braxlosiran (del-brax) Phase 1/2 FORTITUDE program in Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is a genetically acquired disease that leads to progressive muscle weakness and severely decreased functional capacity. The data will be presented at the 32nd Annual FSHD Society International Research is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the disease-causing gene, double homeobox 4 (DUX4). FSHD affects approximately 45,000 to 87,000 people in the United States and Europe. Topline data from these cohorts for del-brax treated participants, compared to placebo, demonstrated: Consistent improvement of functional mobility and muscle strength as measured by 10-meter Walk-Run test (10MWRT), Timed Up and Go (TUG), and quantitative muscle testing (QMT) as compared to placebo; Consistent improvement in multiple measures of quality of life as measured by patient-reported outcomes and compared to placebo; Rapid and significant reductions in levels of KHDC1L and creatine kinase, a biomarker of muscle damage. Favorable long-term safety and tolerability with most adverse events (AEs) mild or moderate, with no related serious or severe adverse events and no discontinuations. Topline data from the ongoing, fully enrolled del-brax Phase 1/2 FORTITUDE biomarker cohort are anticipated in Q2 2026. The primary endpoint of the FORTITUDE biomarker cohort is reduction of KHDC1L, a novel DUX4-regulated circulating biomarker. Avidity also announced that the accelerated approval regulatory pathway in the U.S. is open for del-brax and that the company has initiated the global, confirmatory Phase 3 FORWARD study in FSHD. In a recent research note, Chardan Capital analyst Keay Nakae set a 12-month price target of $75 per share for Avidity Biosciences. The valuation stems from a Net Present Value (NPV) model of the company's projected revenues from its RNA therapeutics pipeline, forecasted out to the year 2034. Chardan maintained its Buy rating and increased its price forecast from $65 to $75. According to Nakae's analysis, the valuation incorporates specific 'probability of success' rates for each of Avidity's drug candidates to account for clinical and regulatory risks. His model assigns a 60% chance of success for the company's FSHD candidate, 50% for its DM1 and DMD exon 44 candidates, and lower probabilities for other assets. The analyst applied a 14% Weighted Average Cost of Capital (WACC) as the discount rate to reflect the high-risk profile of the development-stage company. Nakae also highlighted several significant risks that could impede Avidity from reaching the $75 price target. He noted that the company, which currently has no revenue-generating products, will likely require additional financing, potentially leading to shareholder dilution. The analyst also pointed to the risks of intellectual property challenges, the critical nature of upcoming clinical trial outcomes, and the intense competition from larger, better-resourced companies in the biotechnology sector. Price Action: RNA stock is trading lower by 11.6% to $32.03 at last check Monday. Read Next:Photo by Gorodenkoff via Shutterstock UNLOCKED: 5 NEW TRADES EVERY WEEK. Click now to get top trade ideas daily, plus unlimited access to cutting-edge tools and strategies to gain an edge in the markets. Get the latest stock analysis from Benzinga? AVIDITY BIOSCIENCES (RNA): Free Stock Analysis Report This article Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
