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 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 17, 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
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
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 […]
