Research

Percheron Therapeutics Announces Terminated of ATL1102 Study for DMD

“One way or other, we don’t think ATL-1102 can remain the centerpiece of the story put forward to investors,” Garner told investors in a...

Russian Biotech Company Generium has Begun Clinical Trials of a Gene Therapy for Duchenne Muscular Dystrophy

Clinical studies for a therapy for Duchenne muscular dystrophy have begun at Sirius University and the Russian biotech business Generium. At a gathering at...

Cumberland Pharmaceuticals Announces Fantastic Results from Phase 2 FIGHT Duchenne Trial in DMD Heart Disease

Cumberland Pharmaceuticals, a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from its Phase...

Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201

ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44, was authorized by the UK's Medicines and Healthcare Products Regulatory Agency (MHRA)...

A Novel “Mini-CRISPR” Could Be More Successful at Editing the Dystrophin Gene

Several research groups have been attempting to create compact CRISPR systems that can fit into a single AAV vector while maintaining editing capabilities. The...

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