Jesse's Journey Foundation

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 […]

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 […]

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 […]

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 […]

PTC Therapeutics announced the expansion of its European label for translarna (ataluren). Ataluren restores dystrophin production in individuals with Duchenne caused by a nonsense mutation. This treatment is only available in Europe. The label expansion now allows patients who have lost their ability to walk to continue to use ataluren. Read the full press release […]

Sarepta Therapeutics has completed a new drug submission to the FDA in the United States for Casimersen, their third exon-skipping medicine for individuals with Duchenne amenable to exon 45. Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne. Casimersen is the third exon-skipping medicine using the […]