Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...
Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Clinical Trials Arena on MSN2d
Avidity’s del-zota elicits 25% increase in dystrophin in DMD patientsThe company plans to submit a biologics licence application to the US Food and Drug Administration later this year.
Clinical Trials Arena on MSN3d
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 ...
Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...
This will allow us to gather additional safety data, assess the impact of this dose of PGN-EDO51 on dystrophin levels, and potentially improve the design of CONNECT2," said James McArthur ...
63% of mice treated with two rAAV9-microdystrophin doses produced antibodies to dystrophin, which was less in mice treated with two rAAV9-microdystrophin doses and combination therapy (25%). Likewise, ...
Duchenne muscular dystrophy is a rare, progressive genetic disorder caused by mutations in the DMD gene, which encodes for the dystrophin protein. The absence of functional dystrophin leads to muscle ...
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