June 23, 2020
Since 1995, Jesse’s Journey has strongly believed in strong partnerships with academics and clinicians to fund the most promising research for Duchenne muscular dystrophy. This focus and fundraising effort have brought us to granting more than $13.1 million across 45 research projects. We have attracted applications from leading Duchenne researchers from all over the world including the […]
June 23, 2020
June 16, 2020
Sarepta Therapeutics has announced positive results from their gene therapy, SRP-9001, under investigation for Duchenne muscular dystrophy. Preliminary data based upon four boys demonstrated the therapy to be safe and tolerable at one year in their clinical trial. Results were published in JAMA Neurology and further support the potential for SRP-9001 to provide clinically meaningful […]
June 15, 2020
The European Commission has granted orphan drug designation to viltolarsen, a treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. See the June 8, 2020, vilolarsen press release here. This compound was approved in Japan in March this year under the brand name Viltepso and is currently under priority review by the FDA in the USA. It […]
June 10, 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 […]
June 29, 2020
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 […]
June 26, 2020
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 […]
June 25, 2020
June 24, 2020
On June 23, 2020, Santhera Pharmaceuticals announced the UK’s renewal of its early access program for idebenone, a medication used to treat Duchenne patients who are experiencing respiratory function decline, and not taking glucocorticoids. The UK’s Medicines and Health products Regulatory Agency (MHRA) has recognized the positive risk/benefits of idebenone in Duchenne patients. As a […]
June 17, 2020
