The Speak Foundation

News

• FDA Approves Aquestive Therapeutics’ Exservan for the Treatment of ALS December 6, 2019
  On Nov. 25, the US Food and Drug Administration (FDA) approved riluzole oral film (Exservan) for the treatment of amyotrophic lateral sclerosis (ALS). Exservan is an oral film formulation of riluzole (Rilutek), which has been available in tablet form to treat ALS for more than two decades. Exservan will be made available in the United […]
• Kadimastem Announces Positive Interim Results from Phase 1/2a Study of AstroRx in ALS December 5, 2019
  Israel-based Kadimastem Ltd. announced positive interim results from the first cohort of its phase 1/2a clinical trial evaluating the safety, tolerability, and preliminary efficacy of AstroRx in patients with amyotrophic lateral sclerosis (ALS). Results presented at the 7th International Stem Cell Meeting of the Israel Stem Cell Society held recently in Tel Aviv, Israel, showed that patients […]
• FDA Accepts Roche Genentech’s NDA for Risdiplam for the Treatment of SMA December 3, 2019
  On Nov. 25, Genentech, a member of the Roche Group, announced that the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for risdiplam, an investigational therapy for the treatment of spinal muscular atrophy (SMA). Priority Review status requires the FDA to review the application and decide on whether […]
• Zogenix Announces Positive Results from Pivotal Study of Investigational Treatment MT1621 for TK2 Deficiency December 2, 2019
  At the 24th Annual Congress of the World Muscle Society (WMS) held in October 2019 in Copenhagen, Denmark, Zogenix presented positive results from its pivotal phase 2 retrospective clinical trial (RETRO) testing the safety and efficacy of pyrimidine nucleoside substrate enhancement therapy (SET) MT1621 for treating thymidine kinase 2 (TK2) deficiency. The experimental therapy was developed […]
• Santhera Pharmaceuticals Releases Letter to DMD Community Announcing Temporary Suspension of Enrollment in BreatheDMD Expanded Access Program November 26, 2019
  On Nov. 19, Santhera Pharmaceuticals released a letter to the Duchenne muscular dystrophy (DMD) community announcing its decision to suspend enrollment of new participants in the BreatheDMD Expanded Access Program (EAP) until the SIDEROS clinical trial is fully enrolled. The full letter follows. SIDEROS clinical trial and BreatheDMD program update: Dear Duchenne community, In February 2018, Santhera […]