Neuromuscular Diseases

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Dianthus Therapeutics (DNTH) 2025 Conference Transcript
2025-06-05 14:57
Dianthus Therapeutics (DNTH) 2025 Conference June 05, 2025 09:55 AM ET Speaker0 Good morning, everybody. I'm Marino Garcia, CEO for Dianthus. I will be providing a brief update. Oh, here we go. Maury. How you doing? And then we'll open it up for some Q and A. Before I begin, just again, thank you to Jeffries for this opportunity today. I will be making some forward looking statements, so I urge everyone please to go to our website and where you can read all our SEC filings. This is a very exciting time for ...
Dyne Therapeutics to Present New Preclinical Data in Facioscapulohumeral Muscular Dystrophy at the FSHD Society International Research Congress
GlobeNewswire News Room· 2025-06-05 11:30
Core Insights - Dyne Therapeutics announced new preclinical data for DYNE-302, showing potential functional improvement in facioscapulohumeral muscular dystrophy (FSHD) [1][2] - The data will be presented at the 32nd Annual FSHD Society's International Research Congress in Amsterdam on June 12-13, 2025 [1][4] Company Overview - Dyne Therapeutics is focused on developing therapeutics for genetically driven neuromuscular diseases, leveraging its FORCE™ platform [6] - The company has a broad pipeline that includes clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), as well as preclinical programs for FSHD and Pompe disease [6] Disease Background - FSHD is a rare, progressive genetic disease caused by a mutation in the DUX4 gene, leading to muscle loss and weakness [5] - An estimated 16,000 to 38,000 individuals in the U.S. and approximately 35,000 in Europe are affected by FSHD, with no approved therapies currently available [5] Research Findings - In a mouse model of severe FSHD, a single intravenous dose of DYNE-302 restored the ability to run on a treadmill, indicating potential reversal of muscle damage and inflammation [2][3] - DYNE-302 targets DUX4 mRNA to suppress its expression, aiming to address the underlying cause of muscle damage in FSHD [3] Presentation Details - The oral presentation on DYNE-302's effects will take place on June 13, 2025, at 12:00 p.m. CEST [4] - The presentation will be available on Dyne's website following the session [4]