Jesse's Journey Foundation

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Research Updates

Catabasis Connection: September 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 […]

Santhera Pharmaceuticals: New licensing agreement with ReveraGen Pharma

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 […]

NS Pharma: FDA grants accelerated approval for Viltepso (viltolarsen)

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 […]

Solid Biosciences: Update on SGT-001 Phase 1/2 Clinical Trial

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 Connection: July 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 […]

PTC Therapeutics: Translarna (Ataluren) Update

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: Casimersen Update

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 […]

Santhera Pharmaceuticals: Idebenone for Duchenne Update

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 […]

Sarepta Therapeutics: Positive Safety and Efficacy Data from SRP-9001

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 […]

Nippon Shinyaku: Viltolarsen Update

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 […]