All Duchenne muscular dystrophy (DMD) articles
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NewsFDA hits Sarepta with liver warning labelling for its DMD drug Elevidys
As well as the new safety warnings for the Duchenne muscular dystrophy gene therapy, the biopharma company also faced a new, more restrictive licence for the treatment.
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NewsNovartis strengthens neuroscience pipeline with $12bn Avidity acquisition
Deal to advance potential first-in-class RNA therapeutics focused on genetic neuromuscular diseases such as Duchenne muscular dystrophy.
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Article
Amid FDA turmoil Sarepta’s gene therapy worries begin to ease
But the impact of George Tidmarsh’s CBER appointment after Vinay Prasad’s abrupt resignation remains to be seen.
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NewsDuchenne gene therapy interim trial outcomes “striking”
The gene therapy demonstrated consistent evidence of positively changing disease trajectory in Duchenne muscular dystrophy, new interim data shows.
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NewsCHMP meeting highlights – April 2025
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of a variety of new medicines, including several treatments for hereditary and rare conditions.
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NewsCHMP meeting highlights - June 2024
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended several new medicines, including positive opinions for treatments for rare disorders.
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NewsAAV gene therapy demonstrates positive activity in Duchenne muscular dystrophy
Initial trial results show the gene therapy enabled up to 85 percent of muscle fibres expressing microdystrophin in boys with Duchenne muscular dystrophy eight weeks post-injection.
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OpinionClinical trials for rare diseases
In this interview, Mindy Leffler, Managing Director of Qualitative Research and Psychometrics at Emmes Endpoint Solutions, discusses the nuances of designing clinical trials for rare diseases including specific challenges related to traditional endpoints.
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NewsCHMP meeting highlights: January 2024
In its first meeting of 2024, the CHMP recommended a generic medicine for schizophrenia and refused a marketing authorisation (MA) for geographic atrophy.
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NewsMHRA approves ‘safer’ medicine alternative for rare disease
A new drug has been approved in the UK as a safer alternative to corticosteroids to treat Duchenne muscular dystrophy (DMD).
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NewsCHMP’s September 2023 meeting highlights
In its September 2023 meeting, the EMA’s human medicines committee (CHMP) recommended nine medicines for approval and the extension of therapeutic indications for 11 medicines.
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NewsGene therapy approved for Duchenne muscular dystrophy
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.