A new study in Heart Rhythm, the official journal of the Heart Rhythm Society, the Cardiac Electrophysiology Society, and the Pediatric & Congenital Electrophysiology Society, published by Elsevier, examined… read more.
PTC Therapeutics, Inc. announced that the European Commission (EC) has decided not to adopt the CHMP’s negative opinion of January 24, 2024 on the annual renewal of the… read more.
– Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, announced the FDA has… read more.
Santhera Pharmaceuticals is pleased to note the launch of Agamree (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in the United States by Catalyst Pharmaceuticals, Inc. the… read more.
Novartis presented new data that continue to support the clinical benefits of Zolgensma (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA)…. read more.
Biogen Inc. announced the European Commission (EC) has authorized Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older …. read more.
The European Medicines Agency, the EU drug watchdog, has approved the first medicine using CRISPR/Cas9, a novel gene-editing technology. Casgevy (exagamglogene autotemcel) is indicated for the treatment of… read more.
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