ROSWELL, Ga. and DUBLIN and HAMILTON, Bermuda — Saol Therapeutics, a privately held clinical-stage pharmaceutical company, today announced that the company has been granted a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss new data and a potential path forward for its New Drug Application (NDA)...
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Specific gene profiles of bronchoalveolar lavage (BAL) cells are associated with phenotypic manifestations of sarcoidosis such as activity and disease severity, including immune, inflammatory, and profibrotic mediators as identified via genome-wide transcriptome analysis, according to results published in the European Respiratory Journal. Study results also suggest 4 possible sarcoidosis endotypes with specific...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 (formerly ARO-DM1), an investigational small interfering RNA (siRNA) therapeutic for the treatment of type 1 myotonic dystrophy (DM1). Cohorts...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced screening and enrollment are underway in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to assess prophylactic sirolimus treatment as part of an enhanced safety protocol during treatment...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. We have received feedback from the Medicines & Healthcare products Regulatory Agency (MHRA) in...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to evaluate the use of an enhanced immunosuppressive...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive topline three-year functional results from Part 1-treated patients in EMBARK (Study SRP-9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who...
CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared the first clinical results from two of its siRNA programs for neuromuscular diseases. Early results from Phase 1/2 ascending dose studies of SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003...
CAMBRIDGE, Mass. – Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up. Sarepta Therapeutics touted “statistically significant and clinically meaningful” functional improvements in...
– Treatment with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6) – Safety profile consistent with prior studies of ELEVIDYS and real-world experience CAMBRIDGE, Mass. — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare...