The exon-skipping drug was approved by the FDA for DMD patients with mutations in the dystrophin gene amenable to exon 53 skipping, a group that accounts for around 8% of the total DMD population.
Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping ...
Clinical Trials Arena on MSN6d
Wave Life Sciences unveils Phase II data for DMD therapy, WVE-N531Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...
It would be able to target the DMD patients who are not receiving exon skipping drugs due to weekly dosing being needed. Plus, such drugs already approved are providing limited efficacy.
Clinical Trials Arena on MSN16d
Dyne’s DMD trial advances patient mobility and dystrophin expressionThe Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an increase over baseline at 12 months.
ENTR-601-44 is an investigational therapy for the potential treatment of people living with DMD who are exon 44 skipping amenable. The therapy is being evaluated for its potential to “skip” over the ...
The trial has enrolled a number of boys and young men with DMD who are amenable to exon-51 skipping. CONNECT2 is a 25-week, multinational, double-blind, placebo-controlled trial with multiple ...
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