Duchenne muscular dystrophy (DMD)

news

Treatment for rare Duchenne muscular dystrophy mutation approved by FDA

26 February 2021 | By Hannah Balfour (European Pharmaceutical Review)

The FDA conditionally approved Sarepta’s Amondys 45 (casimersen) after interim Phase III results indicated the treatment is reasonably likely to be of clinical benefit.

news

Positive results in Duchenne muscular dystrophy (DMD) trial

29 September 2020 | By Hannah Balfour (European Pharmaceutical Review)

Interim trial results suggest vamorolone may offer a superior alternative to long-term treatment with high-dose glucocorticoids in young Duchenne muscular dystrophy (DMD) patients.

article

Emerging biotechs tackle medical research continuity concerns amid COVID-19

24 April 2020 | By Rich Horgan (Founder and President of Cure Rare Disease)

While medical professionals fight COVID-19 on the front line, small biotech businesses face a new uphill battle – maintaining research continuity for non-COVID-19 life-changing therapeutics.

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FDA grants accelerated approval for treatment of rare DMD mutation

16 December 2019 | By Hannah Balfour (European Pharmaceutical Review)

The FDA has granted accelerated approval to Vyondys 53 for the treatment of patients with Duchenne muscular dystrophy containing a mutation of the dystrophin gene that is amenable to exon 53 skipping.

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Complete response letter handed to Sarepta Therapeutics

20 August 2019 | By Victoria Rees (European Pharmaceutical Review)

The pharmaceutical company has received a complete response letter for its golodirsen injection, which had been submitted for accelerated approval.