Translarna (Ataluren) Rejected by EMA: Marketing Authorization Not Renewed
In May 2024, PTC Therapeutics had been announced that the European Commission (EC) has decided not to adopt the CHMP's negative opinion on the...
Wave Life Sciences Announces Positive Data from FORWARD-53 Clinical Trial in DMD Including Significant Functional Benefit and Reversal of Muscle Damage after 48 Weeks...
Wave Life Sciences, a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced positive data...
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201
Entrada Therapeutics, announced it has received authorization from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee to initiate...
Regenxbio Reports Positive Biomarker Data From Affinity Duchenne Trial Of Rgx-202 Gene Therapy
REGENXBIO reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated...
Satellos Presents Initial Data from the Phase 1 Trial of SAT-3247 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference
Satellos Bioscience, a biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced initial Phase...