DMD News Briefs

Repository for DMD and BMD Introduced at NORD Breakthrough Summit

A decentralized platform connecting researchers to coded data and samples from people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) was presented in a poster at the National Organization for Rare Disorders (NORD) Breakthrough Summit 2021. The platform, called CureDuchenne Link, is a data hub that connects the information provided by DMD and…

DMD

Experimental Gene Therapy Improves Motor Ability in DMD

SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) is well tolerated and leads to an overall improvement in motor ability compared to baseline in patients with Duchenne muscular dystrophy (DMD), according to results from the SRP-9001-101 study reported at the World Muscle Society (WMS) 2021 Virtual Congress. These improvements were maintained over 3 years, meaning the response was durable. Roche and…

Fenofibrate Degrades Myostatin and Helps Protect Muscles in DMD Models

Fenofibrate showed therapeutic effects in Duchenne muscular dystrophy (DMD) models, according to a new study published in the British Journal of Pharmacology. During the study, treatment with fenofibrate improved muscle function and reduced muscle damage in mdx mice, the most common DMD animal model. The study showed that fenofibrate inhibited the expression of myostatin protein…

First Genetic Identifier for Autism Found

Gene Therapy Offers Promise in Treating Duchenne, Along with Significant Obstacles

By 2025, between 10 and 20 new gene therapies could win approval annually by the US Food and Drug Administration (FDA), most of them delivered via adeno-associated virus (AAV).  Yet Duchenne muscular dystrophy (DMD) presents some unique challenges for AAV-delivered gene therapy — namely because the dystrophin gene, which is missing in DMD, is one of…

DMD

ADAMTS1 Could Be New Therapeutic Target in DMD

Anti-ADAMTS1 antibody relieved muscle dysfunction and fibrosis in a mouse model with Duchenne muscular dystrophy (DMD), found in a new study by Chinese researchers published in the journal Life Sciences. This finding suggests ADAMTS1 could be a new therapeutic target for the disease. ADAMTS1 is a metalloproteinase that was reported to be highly expressed in…

Genetic research

A New Approach for Correcting Diverse DMD Mutations

Researchers have developed homology-independent targeted integration (HITI)-mediated gene editing to correct full-length human dystrophin in Duchenne muscular dystrophy (DMD). The authors of this new method observed full-length dystrophin restoration in skeletal and cardiac muscles of a humanized mouse model of DMD, the hDMDΔ52/mdx model, recently published in Molecular Therapy. The mice contained a full-length human…

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