"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...
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Precision BioSciences reports encouraging muscle improvements in preclinical DMD gene therapy study
Precision BioSciences Inc. (NASDAQ:DTIL) presented new preclinical findings for its PBGENE-DMD gene therapy at the Muscular ...
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...
Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...
Edgewise's Phase 2 trial for sevasemten in Becker muscular dystrophy showed a 28% average CK reduction vs. placebo (p=0.02). Functional measures, including NSAA and timed walking tests, showed ...
Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular ...
"The incidence of DMD in our study was similar to that reported in previous studies . . . Overall, our results indicate that newborn screening for DMD is feasible, and this will be good news for the ...
Early screening for Duchenne muscular dystrophy can help you get early treatments and referrals for your child to help prevent further loss of muscle tissue. Duchenne muscular dystrophy (DMD) is a ...
Avidity Biosciences, Inc. (NASDAQ:RNA) on Wednesday shared new data from participants treated continuously with del-zota for one year in the EXPLORE44 and EXPLORE44-OLE trials for Duchenne muscular ...
The X-linked neuromuscular disorder Duchenne muscular dystrophy (DMD), which historically has been diagnosed in boys between the ages of 3 and 6 years, has become an important candidate for newborn ...
Melanie Sanford was worried that her son, Hudson, wasn’t jumping around as much as other toddlers his age. But his pediatrician reassured her that kids all develop at their own pace, and “he wasn’t ...
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