A natural history study of limb girdle muscular dystrophy type 2A (LGMD2A, a form of calpainopathy) has been initiated by Linda Lowes, PT, PhD at Nationwide Children’s Hospital in Columbus, Ohio. The aim of this study is to observe
Myonexus Therapeutics announces initiation of a gene therapy clinical trial for LGMD2E
On November 8, 2018, Myonexus Therapeutics announced the initiation of a clinical trial evaluating MYO-101, a novel gene therapy candidate for beta-sarcoglycanopathy, also known as limb girdle muscular dystrophy type 2E (LGMD2E). The trial is underway at Nationwide Children’s Hospital’s
FDA to step up enforcement of stem cell clinics
Stem cells are cells that have the unique ability to self-renew and to transform into many specialized cell types. They can be isolated either from embryos or from adult tissue. Adult stem cells are being used for various medical therapies
Rare disease stakeholders connect at 2017 RARE Patient Advocacy Symposium
Rare disease patients, caregivers, representatives from advocacy organizations, and other rare disease stakeholders met on May 19, 2017 in Philadelphia for the 2017 RARE Patient Advocacy Symposium, a partnership of Global Genes and the Penn Medicine Orphan Disease Center. The
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