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 ...
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Precision BioSciences Announces Oral Presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Presentation Details: Title: ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Del45-55 Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD Date and Time ...
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 ...