– EU label update supports Translarna use in patients who became non-ambulatory while on therapy – SOUTH PLAINFIELD, N.J., June 29, 2020 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee […]
06/26/20 8:30 AM EDT — Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne — — Casimersen is the third exon-skipping […]
06/15/20 11:00 AM EDT — Results at one year demonstrate continued safety and tolerability of SRP-9001 micro-dystrophin gene therapy in four patients with Duchenne muscular dystrophy — — Confirmed vector […]
Kyoto, Japan, June 8, 2020 – Nippon Shinyaku Co., LTD. (Nippon Shinyaku; Kyoto; President, Shigenobu Maekawa) announced that European Commission (EC) has granted Orphan Drug Designation to viltolarsen (NS-065/ NCNP-01) […]
Pratteln, Switzerland, June 2, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces that partner ReveraGen Biopharma Inc. has completed a long-term, open-label extension study of 24 months duration with vamorolone in […]
May 20, 2020 Dear Duchenne community, As you know from our previous updates, Santhera’s main focus in the U.S. in the first half of the year has been to bring […]
Friday, May 15, 2020 – 8:00amEDT Pfizer Inc. (NYSE: PFE) today announced updated Phase 1b clinical data on PF-06939926, an investigational gene therapy being developed to treat Duchenne muscular dystrophy […]
In this month’s Catabasis Connection, they discuss: Learning in their Phase 3 PolarisDMD trial Boys with Duchenne demonstrate capsule swallowing abilities The expected patient population Safety data on edasalonexent.
— The Interim data from EPYDIS Phase 3 trial shows that the trial is NOT FUTILE and the Independent Data Monitoring Committee recommends trial continuation — — Blinded re-estimation of […]
