Duchenne muscular dystrophy

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Capricor files cell therapy for DMD cardiomyopathy with FDA

Capricor completes its US filing for deramiocel, vying to become the first FDA-approved therapy for cardiomyopathy in Duchenne muscular dystrophy.

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Price deal opens door to NHS use of Santhera DMD drug

Santhera's Duchenne muscular dystrophy (DMD) therapy Agamree has been cleared for NHS use after a confidential pricing agreement.

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Gene therapy firm Kate Tx snapped up by Novartis for $1.1bn

Novartis buys gene therapy specialist Kate Therapeutics for $1.1 billion, adding an early-stage rival to Sarepta's muscular dystrophy therapy Elevidys

News

Sarepta pulls plug on Duchenne exon-skipping drug

Sarepta Therapeutics has decided not to continue the development of vesleteplirsen, a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51.

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Surfing on new Duchenne data, Wave charges $175m offering

Phase 2 results with Wave Life Sciences' Duchenne muscular dystrophy candidate WVE-N531 pushed the company's share price to its highest level in a year and could lead to discussions with re

News

Buoyed by muscular dystrophy data, Avidity raises $345m

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy, the biotech has closed a public offering that raised an imp

Capricor files cell therapy for DMD cardiomyopathy with FDA

Price deal opens door to NHS use of Santhera DMD drug

Gene therapy firm Kate Tx snapped up by Novartis for $1.1bn

Sarepta pulls plug on Duchenne exon-skipping drug

Surfing on new Duchenne data, Wave charges $175m offering

Buoyed by muscular dystrophy data, Avidity raises $345m

Sling shoots at thyroid eye disease with its oral therapy

Health Trends 2025: Reimagining What's Possible

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Duchenne muscular dystrophy

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