Precision BioSciences presents preclinical data from PBGENE-DMD program
Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...
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Avidity’s del-zota elicits 25% increase in dystrophin in DMD patients
The company plans to submit a Biologics Licence Application to the US Food and Drug Administration later in 2025 for its DMD ...
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Avidity’s del-zota elicits 25% increase in dystrophin in DMD patients
Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results from the company’s ...
Roche To Share Latest Scientific Advancements From Its Neuromuscular Portfolio At Muscular Dystrophy Association (MDA) 2025 Conference
New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ind ...
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Dyne Therapeutics Reports Promising Long-Term Data for DYNE-251 in Duchenne Muscular Dystrophy with Plans for Accelerated Approval Submission
In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44. About Duchenne Muscular Dystrophy (DMD) DMD is a rare ...
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Buoyed by muscular dystrophy data, Avidity raises $345m
Avidity is hoping to follow in the footsteps of Sarepta, which has launched a series of exon-skipping therapies for DMD, targeting exons 51, 53, and 45, that brought in around $1 billion in sales ...