May 15, 2020
On May 15, 2020, Pfizer announced positive results from their phase 1B clinical trial on PF-06939926, an investigational gene therapy under development for Duchenne muscular dystrophy. Preliminary data for 9 boys, still walking, aged 6-12, showed encouraging benefits on a number of parameters with a manageable safety profile. There were a few serious adverse events […]
May 13, 2020
On May 13, 2020, Solid Biosciences announced an update on their phase 1/2 clinical trial on SGT-001, an investigational gene therapy under development for Duchenne. This trial is currently on hold due to some safety concerns, but patients enrolled are continuing to engage in study-related activities. Their clinical biomarker results presented at a recent scientific […]
May 13, 2020
Exciting news for Capricor’s CAP1002 therapy for the treatment of cardiomyopathy in advance stage Duchenne muscular dystrophy: On May 13, 2020, Capricor announced positive top-line results from their HOPE-2 study with their lead candidate CAP-1002 treating Duchenne patients with an advanced stage of the disease. CAP1002 is a cardiac-derived cell therapy currently under investigation for […]
May 12, 2020
Due to COVID-19, the Defeat Duchenne Family Forum presented by PTC Therapeutics and in partnership with La Force DMD has been transformed from a one-day “in-person” program into a series of four virtual events taking place May 20 – 30, 2020. Join us as families and caregivers navigating the Duchenne journey across Canada come together with researchers, clinicians, and […]
May 6, 2020
May 12, 2020
Catabasis has provided updates on edasalonexent and data from three scientific posters shared during the Muscular Dystrophy Association (MDA) Virtual Poster Session. Click here to see the May 2020 – Catabasis Connection Edasalonexent: In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle […]
May 12, 2020
The Walk to Defeat Duchenne presented by Sbenati Dentistry has been rescheduled to Monday, September 7, 2020 (World Duchenne Awareness Day) – this means more time to grow your team and raise vital funds! While making plans during this uncertain time is challenging, we hope you will refocus your efforts online – by encouraging your supporters to […]
April 28, 2020
Italfarmaco has provided an update regarding the Givinostat clinical trial for the Duchenne community: 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 the study sample size allows a reduction of the total number of subjects to […]
March 30, 2020
Catabasis has shared its March 2020 update: Catabasis provided an update regarding COVID-19 and Duchenne, FAQ’s, and shared the presentation slides from the MDA Virtual Clinical Trials Session. March 2020 – Catabasis Connection Issue 25 Edasalonexent Update: In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of […]
March 24, 2020
Santhera Pharmaceuticals shared its 2019 Annual Results and Highlights Piepleine Progress for Duchenne muscular dystrophy. “Santhera is emerging as a leader in addressing rare neuromuscular diseases and 2019 saw us prepare the business for several significant value inflection points this year for our drug candidatesto treat DMD,” said Dario Eklund, CEO of Santhera. “The regulatory […]
