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Babies with SMA able to swallow with Evrysdi® treatment
All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
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Duchenne muscular dystrophy (DMD)
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First UK patient enrolled in Duchenne muscular dystrophy gene therapy trial
The Phase III trial will evaluate the safety and efficacy of Pfizer’s PF-06939926 gene therapy in 99 paediatric Duchenne muscular dystrophy patients across 15 countries.
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Treatment for rare Duchenne muscular dystrophy mutation approved by FDA
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.
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Positive results in Duchenne muscular dystrophy (DMD) trial
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.
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Emerging biotechs tackle medical research continuity concerns amid COVID-19
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
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
The pharmaceutical company has received a complete response letter for its golodirsen injection, which had been submitted for accelerated approval.
