Baby gene therapy study offers hope for fatal muscle disease – WRAL.com
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Baby gene therapy study offers hope for fatal muscle disease
WRAL.com WASHINGTON — A first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe has extended the tots’ lives, and some began to roll over, sit and stand on their own, researchers reported Wednesday. Only … |
Rare Disease Report (blog) |
Sarepta Investing in CRISPR Technology to Develop DMD Drugs
Rare Disease Report (blog) “Although early, CRISPR technology represents hope for a large percentage of individuals with DMD. Excising certain exons has the potential to correct a majority of DMD mutations. Toward that goal, we’ve shown in mouse models that we can excise exons … Sarepta Therapeutics, Inc. (SRPT) registers a price change of 2.84% while CRISPR Therapeutics AG (CRSP) finishes …Stocks Gallery Sarepta and Duke University Collaborating on Gene Editing as a Treatment for Duchenne MDMuscular Dystrophy News |