24 Mar
  • By Brandon
  • Cause in

Santhera Reports 2019 Annual Results and Highlights Pipeline Progress in DMD

Santhera Pharmaceuticals is reporting revenues slightly above what they expected for 2019 and is clearly focused on DMD treatments as a high priority for 2020: Puldysa (idebenone) and vamorolone are both making progress. Puldysa is being studied in the Phase 3 SIDEROS trial for the treatment of respiratory dysfunction in patients with DMD who are […]

READ MORE
09 Mar
  • By Brandon
  • Cause in

NS Pharma is launching expanded access for viltolarsen

CureDuchenne is excited to share that NS Pharma is launching expanded access for viltolarsen, allowing patients with DMD amenable to exon 53 skipping to receive this product while it is being reviewed by the FDA. CureDuchenne thanks NS Pharma for showing dedication to the DMD community by opening access to this treatment merely weeks after FDA’s […]

READ MORE
24 Feb
  • By Brandon
  • Cause in

PFIZER INITIATES PHASE III DUCHENNE GENE THERAPY CLINICAL TRIAL

CureDuchenne congratulates Pfizer on their progress to a Phase III clinical trial for #PF-06939926, its AAV9 mini-dystrophin gene therapy for Duchenne muscular dystrophy. We’re grateful to Pfizer for their dedication to the Duchenne community. Pfizer’s announcement is a big step forward for gene therapy efforts in Duchenne, an innovative field where there is still so […]

READ MORE
08 Jan
  • By Greg Freeman
  • Cause in

Italfarmaco – Winter Newsletter 2020

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 us in Houston, Texas at our CureDuchenne Cares Workshop on February 8, 2020. Two great reasons to sign up for the Workshop: Italfarmaco will be there […]

READ MORE
19 Dec
  • By Greg Freeman
  • Cause in

Mallinckrodt Comments on its BRAVE study

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 to 8 Years of Age With Duchenne Muscular Dystrophy) DECEMBER 19, 2019 -(MALLINCKRODT)- After careful consideration and despite engaging in diligent efforts to research, develop […]

READ MORE
15 Oct
  • By Greg Freeman
  • Cause in

cTAP Publishes Comprehensive Analysis of the North Star Ambulatory Assessment (NSAA), a Newly Adopted Primary Outcome Measure in Clinical Trials of Patients with Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.–(BUSINESS WIRE)–The collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, global coalition in Duchenne muscular dystrophy (DMD), publishes research in the journal PLOS ONE providing quantitative insights to assist the design and analysis of clinical trials in patients with DMD. The NSAA outcome measures the ability of patients to conduct a variety of activities – […]

READ MORE