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 […]
Italfarmaco’s EPIDYS clinical trial for Givinostat is enrolling boys age 6 and up at 40 sites around the world. See the details in Italfarmaco’s Winter 2020 community newsletter. Or join […]
Mallinckrodt Comments on its BRAVE study (A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 […]
