September 24, 2020
On September 22, 2022, Santhera announced its publication of long-term data with vamorlone in Duchenne muscular dystrophy. This is data extended from their previously published 24-week data and continues to show a reduction in steroid associated safety issues such as stunting of growth, cushingoid appearance (rounded face), hirsutism (excessive hair growth), weight gain and behavioural […]
September 21, 2020
Catabasis has provided an update on how they’re preparing to share top-line results from the Phase 3 PolarisDMD trial. Click here to see the September 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 disease progression. By […]
September 10, 2020
September 9, 2020
On September 2, 2020, Santhera announced their new licensing agreement with ReveraGen Pharma, the company up until now, responsible for vamorolone. This agreement offers Santhera the advantage of full control of vamorolone. It allows them to pursue the development, registration, and commercialization of this anti-inflammatory drug candidate more effectively and efficiently. Santhera remains committed to […]
August 20, 2020
The FDA has granted accelerated approval for Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene is amenable to exon 53 skipping. Click here to read the full press release. The early clinical data demonstrated an increase in dystrophin production that is reasonably […]
August 19, 2020
August 17, 2020
July 24, 2020
Jesse’s Journey would like to congratulate Dr. Michael Rudnicki, Chief Scientific Officer of Satellos Bioscience and a Jesse’s Journey research funding recipient, for being elected as a Fellow of the Royal Society, the United Kingdom’s national academy of sciences. The Royal Society is the world’s oldest and most esteemed scientific institution founded in 1660 and […]
July 24, 2020
On July 24, 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 […]
July 23, 2020
Catabasis has provided an update on their “North Star Ambulatory Assessment in Duchenne Muscular Dystrophy” as presented at the Parent Project Muscular Dystrophy (PPMD) Conference in July 2020. Click here to see the July 2020 – Catabasis Connection Edasalonexent: In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key […]
