Pamrevlumab for the treatment of DMD
FibroGen receives Fast Track designation from the U.S. FDA for Pamrevlumab to treat Duchenne muscular dystrophy (DMD). April 12, 2021, Original press release. Source: FibroGen, Inc FibroGen, Inc. announced […]
Author Archive for: Marie
Entries by Marie-Catherine Du Berger
Solid Biosciences reports efficacy and safety data
Solid Biosciences reports efficacy and safety data from the ongoing IGNITE DMD clinical trial and resumption of patient dosing in the 2E14 VG/kg cohort. La Force is sharing this press […]
Article about DMD in Nature.com
Recent studies on Duchenne muscular dystrophy (DMD) have greatly deepened our understanding of the primary and secondary pathogenetic mechanisms. Guidelines for the multidisciplinary care for DMD that address obtaining a […]
News from Vamorolone in Duchenne Muscular Dystrophy
Santhera Announces Completion of First 6-Month Period of Pivotal VISION-DMD Trial with Vamorolone in Duchenne Muscular Dystrophy Original Press release > Pratteln, Switzerland, March 3, 2021 Santhera Pharmaceuticals announces that […]
FDA Approval of AMONDYS 45 (casimersen)
Sarepta Therapeutics announces FDA approval of AMONDYS 45 (casimersen) injection to treat Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. Feb. 25, 2021, >Original press release< Sarepta […]
Italfarmaco provides an update on Givinostat
Italfarmaco provides an update on Givinostat, an ongoing clinical program, in an oral presentation at XVIII International Conference on Duchenne and Becker Muscular Dystrophy. Feb. 22, 2021, > Original press release […]
Rare disease day 2021 – La Force DMD
We wish to emphasize that the information contained in this article comes mainly from the RQMO website, the CORD website and the Rare Disease Day website. We thank them. February 28th is Rare disease day 2021. […]
Sarepta announces top-line results for its investigational gene therapy
Sarepta Therapeutics announces top-line results for part 1 of study 102 evaluating SRP-9001, its investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD) Jan. 7, 2021, >Original […]
Pfizer doses the first participant with Its gene therapy
Pfizer doses the first participant in phase 3 study for Duchenne muscular dystrophy (DMD) investigational gene therapy. January 07, 2021, > Original press release < Pfizer Inc. announced that the […]
Positive clinical results from MOMENTUM
Sarepta Therapeutics announces positive clinical results from MOMENTUM, a phase 2 clinical trial of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51. Original press release > […]
