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706,821 Results for "dmd therapeutics inc".
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Duchenne muscular dystrophy
5 DMD Candidates to Watch in 2025
Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and more aim to deliver the next wave of progress with near-term data and regulatory milestones.
January 27, 2025
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6 min read
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Kate Goodwin
Duchenne muscular dystrophy
Dyne Touts ‘Unprecedented’ Dystrophin Expression in Phase I/II DMD Trial for Exon-Skipping Therapy
The highly anticipated results come as the company makes significant changes to its C-suite. Despite the turnover, Dyne said it is looking toward expedited approval pathways for its DMD treatment.
September 3, 2024
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3 min read
·
Tristan Manalac
Duchenne Muscular Dystrophy
Déjà Vu for PepGen as FDA Places Hold on DMD Drug
The FDA has put a stop to U.S. initiation of PepGen’s Phase II trial for its Duchene muscular dystrophy treatment. The company faced the same hurdle for an earlier neuromuscular candidiate in 2023.
December 16, 2024
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2 min read
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Annalee Armstrong
Press Releases
Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD)
January 27, 2025
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11 min read
BioCapital
REGENXBIO to Participate in Cantor Fitzgerald’s Virtual DMD and Other Dystrophy Days
REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in Cantor Fitzgerald’s Virtual DMD and Other Dystrophy Days on Wednesday, April 3, 2024.
March 27, 2024
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1 min read
Duchenne muscular dystrophy
Duchenne Muscular Dystrophy Space on Cusp of Pivotal Era
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
January 27, 2025
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8 min read
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Kate Goodwin
Duchenne muscular dystrophy
Regenxbio Gears Up to Challenge Sarepta in DMD as Gene Therapy Advances to Pivotal Studies
Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.
November 19, 2024
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2 min read
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Tristan Manalac
Earnings
Sarepta Halts Development of Next-Gen DMD Drug, Reports Robust Elevidys Sales
The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.
November 7, 2024
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2 min read
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Heather McKenzie
Press Releases
ImmunoForge Receives IND Approval for Phase 2 Clinical Trial of ‘Pemziviptadil’ for DMD Cardiomyopathy Treatment from FDA, and KF1601, a chronic myeloid leukemia treatment, phase 1 clinical trial IND approved by MFDS
January 14, 2025
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5 min read
Catalyst Pharmaceuticals Announces AGAMREE® Now Commercially Available in the U.S. for the Treatment of Duchenne Muscular Dystrophy (DMD)
Catalyst Pharmaceuticals, Inc. today announced the U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40 mg/mL for the treatment of Duchenne Muscular Dystrophy (“DMD”) in patients aged two years and older.
March 13, 2024
·
7 min read
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