Duchenne muscular dystrophy
| 4 min read
Data on a new oral pill for Duchenne muscular dystrophy, a miss for a heart drug, a $10B acquisition of Crinetics, and more led the news this week.
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FDA updates, pharma tariff agreements, gene editing deals, and more led the news this week.
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The HDAC inhibitor givinostat has received regulatory approval in the US, EU, and UK for patients six years and older.
| 6 min read
Avidity remains on track to submit Biologics License Application at the end of 2025 for accelerated approval
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Long-term treatment with givinostat can significantly delay loss of key mobility functions in patients with DMD
| 3 min read
This recognition signals momentum for next-gen RNA therapeutics designed to enhance delivery and efficacy in treating rare genetic diseases.
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Another death linked to Sarepta’s gene therapy, a promising glioblastoma trial, a rejection for Replimune, and more led the news this week.
| 3 min read
A blood test for organ rejection, another death related to gene therapy for Duchenne muscular dystrophy, a setback for AbbVie, and more led the news this week.
| 5 min read
A groundbreaking approach targets muscle stem cells to restore regeneration in Duchenne muscular dystrophy, offering new hope beyond traditional dystrophin-focused therapies.
| 3 min read
New dual AAV vector delivery overcomes challenges in delivering large genes.
