Shares of Dyne Therapeutics gained after the company shared positive data from a continuing trial of a potential Duchenne muscular dystrophy treatment. The stock rose 14%, to $16.93, midday Monday.
ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced the presentation of ten abstracts at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference ...
"Fast Track designation is an important regulatory milestone for PBGENE-DMD and reflects the significant unmet need in DMD," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "We ...
New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history -- Data expand ...
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Can Keros Therapeutics Rinvatercept stand out in the DMD market?
Keros Therapeutics KROS is focused on developing and commercializing novel therapeutics to treat a wide range of patients ...
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Precision BioSciences shares jump after FDA grants fast track status to Duchenne therapy
Shares of Precision BioSciences (NASDAQ:DTIL) rose 13% on Monday after the U.S. Food and Drug Administration granted Fast Track designation to PBGENE-DMD, the company’s gene-editing therapy for ...
Dyne Therapeutics’ experimental treatment for Duchenne muscular dystrophy (DMD) showed promise in new data from a small Phase 1/2 trial dubbed DELIVER, the pharma announced this week. The drug, ...
Panelists discuss how the pooled 3-year data for delandistrogene moxeparvovec show sustained motor function stabilization and less deterioration compared with natural history, providing confidence in ...
Solid Biosciences (SLDB) is hoping to advance a next-generation gene therapy for the treatment of patients with Duchenne Muscular Dystrophy [DMD]. This would be with a gene therapy candidate by the ...
Experts discuss the landscape of treatment options for Duchenne muscular dystrophy, examine the benefits and risks of gene therapy and conventional steroid treatments, and emphasize the need for early ...
Sarepta Therapeutics said it will update its prescribing information for Elevidys ® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...
Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...
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