Latest news with #myotonicdystrophy
Yahoo
12 hours ago
- Business
- Yahoo
Dyne Therapeutics to Host Investor Conference Call and Webcast to Provide Update on DYNE-101 for Myotonic Dystrophy Type 1, Tomorrow Tuesday, June 17 at 8:00 a.m. ET
WALTHAM, Mass., June 16, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage neuromuscular disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced it plans to provide an update on DYNE-101 in myotonic dystrophy type 1 (DM1) tomorrow, June 17, 2025, and to host a webcast at 8:00 a.m. ET. The company intends to issue a press release prior to the start of the event. Investor Conference Call and Webcast The live webcast will be available on the Events & Presentations page of the Investors & Media section of Dyne's website, and a replay will be accessible for 90 days following the presentation. An accompanying slide presentation will also be available. To view the live webcast and replay, please visit About Dyne Therapeutics Dyne Therapeutics is discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that are designed to overcome limitations in delivery to muscle tissue and the central nervous system (CNS). Dyne has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD). For more information, please visit and follow us on X, LinkedIn and Facebook. Contacts:InvestorsMia Tobiasir@ 781-317-0353 MediaStacy Nartkersnartker@ 781-317-1938
Yahoo
29-05-2025
- Business
- Yahoo
ARTHEx Biotech to Participate in the 2025 Jefferies Global Healthcare Conference
VALENCIA, Spain, May 29, 2025 /PRNewswire/ -- ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, announced today its participation in the 2025 Jefferies Global Healthcare Conference, being held June 3-5, 2025. Frédéric Legros, Executive Chairman and CEO, will participate in one-on-one meetings with investors during the conference. About ARTHEx BiotechARTHEx Biotech is a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression. The Company's lead investigational compound, ATX-01, is being evaluated for the treatment of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder, in the Phase I-IIa ArthemiR™ trial. ARTHEx is also advancing its in-house discovery engine to identify and develop microRNA modulators for other disorders with high unmet medical needs, including genetically-driven diseases like DM1. The Company headquarters are in Valencia, Spain. For more information, please visit and engage with us on LinkedIn. Company Contact. Investor and Media Contact Frédéric LegrosExecutive Chairman and CEOflegros@ +33679495790 Amy ConradJuniper Pointamy@ +1 858-366-3243 View original content to download multimedia: SOURCE ARTHEx Biotech 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
Reuters
29-05-2025
- Business
- Reuters
PepGen to discontinue development of experimental Duchenne therapy; shares drop
May 28 (Reuters) - Drug developer PepGen (PEPG.O), opens new tab said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in a mid-stage study. Shares of the company were down 9.5% at $1.43 in extended trading. The therapy PGN-EDO51 did not achieve target levels of dystrophin production. Dystrophin is a protein whose absence leads to muscle weakening in patients with DMD. DMD affects an estimated one in 3,500 male births worldwide, according to the National Organization for Rare Disorders. It weakens skeletal and heart muscles, deteriorating them quickly over time, and patients often die by the time they are 25. The company said it intends to wind down all DMD-related research and development activities, shifting its focus to a therapy that is being developed for a type of myotonic dystrophy, another muscle wasting disorder. PepGen expects to report data from an early-stage study in myotonic dystrophy in the second half of the year, and a mid-stage study in the first quarter of 2026.
