Scribe Therapeutics and Prevail Therapeutics to Share In Vivo CRISPR-based Genome Editing Data at ASGCT 2025
Scribe Therapeutics, a genetic medicines company unlocking the potential of CRISPR to transform human health, today announced its participation in the 28th American Society...
Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy
Capricor Therapeutics , a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the completion of a...
Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy
Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated...
Agamree (vamorolone) will be Covered in Slovenia by the Slovenian Health Insurance Fund
The Slovenian Health Insurance Institute (ZZZS) today added Agamree (vamorolone) to the list of drugs for the treatment of Duchenne muscular dystrophy in patients...
Russia-Based Circle of Kindness Foundation Announces 18% Price Cut for Elevidys Gene Therapy
According to the Foundation's press office, "as a result of the negotiations, the price of one of the most expensive drugs (Elevidys) in the...