News & Updates | Duchenne Muscular Dystrophy

News & Resources

Santhera and ReveraGen: Successful FDA Pre-NDA Meeting for Vamorolone

November 18, 2021

Jesse's Journey is pleased to share that Santhera Pharmaceuticals and ReveraGen BioPharma have reached an important regulatory milestone for Vamorolone with the U.S. Food and Drug Administration (FDA). There was a successful pre-New Drug Application (NDA) meeting to obtain agreement from the FDA on the sufficiency and adequacy of the clinical data to support an [...]

Solid Biosciences: Letter to the Duchenne Community

November 16, 2021

Jesse's Journey is pleased to share the latest Duchenne community letter from Solid Biosciences. The letter outlines recent functional data from the ongoing IGNITE DMD Phase I/II clinical trials and plans for the dosing of additional patients by the end of the year. We will continue to monitor the progress of this trial and work [...]

Edgewise Therapeutics: Positive Topline Results from EDG-5506 Phase 1 Study

November 1, 2021

Edgewise Therapeutics Announces Positive Topline Results from the EDG-5506 Phase 1 Multiple Ascending Dose (MAD) Study in Healthy Volunteers (HVs) and Doses First Becker Muscular Dystrophy (BMD) Patients Jesse’s Journey is pleased to share news from Edgewise Therapeutics regarding positive top-line results from their EDG-5506, a small molecule myosin inhibitor, Phase 1 Study in Duchenne [...]

Sarepta: Launch of EMBARK – Gene Therapy Study

October 5, 2021

Jesse’s Journey is pleased to share with our Canadian Duchenne community the latest release from Sarepta Therapeutics. They recently announced the initiation of EMBARK and the advancement of the SRP-9001 program. This represents the first pivotal double-blind gene therapy trial in Duchenne, initiated in the US, Europe and Asia. Doug Ingram, CEO and president, said: [...]

Pfizer: Change in Gene Therapy Protocol

September 29, 2021

Pfizer has announced a change to its gene therapy clinical program. Pfizer’s investigational gene therapy trial started recruitment in Canada in March 2021. They recently announced they will be changing their trial protocol to exclude patients with any mutation (exon deletion, duplication, insertion, or point mutation) affecting exons 9-13, or deletions that affect both exon [...]

Warning on Stem Cell Therapy in Duchenne

November 3, 2021

Jesse’s Journey is sharing an important announcement from the World Duchenne Organization and Treat-NMD with our Canadian Duchenne community: The World Duchenne Organization is expressing its concerns regarding an organization that claims to be raising money for a ‘lifesaving’ stem cell treatment. We are supporting and sharing the statement from Treat-NMD, the organization that represents [...]

Research Grant Recipient: Satellos Bioscience

September 8, 2021

Jesse's Journey is proud to announce a research partnership and infrastructure grant to support the development of Satellos' novel approach to treating Duchenne. Satellos scientists are developing small molecule drugs that they believe will restore faulty regeneration and repair observed in the muscles of patients with Duchenne and potentially other degenerative muscle disorders. The company's drug [...]

Health Canada: National Strategy for Drugs for Rare Diseases Update

July 27, 2021

In January 2021, Health Canada launched a national online initiative to engage Canadians in the development of a National Strategy for Drugs for Rare Diseases. More than 650 patients, families, stakeholders, and patient organizations - like Jesse's Journey - provided their diverse perspectives on getting better access to the effective drugs they need. Throughout the [...]

NS Pharma: VILTEPSO® (viltolarsen) Injection Long-Term Efficacy and Safety Data

July 6, 2021

N.S. Pharma, Inc. has announced new long-term efficacy and safety data from the open-label extension trial of a Phase 2 study of VILTEPSO® (viltolarsen) injection. VILTEPSO is an exon-skipping therapy for individuals with Duchenne muscular dystrophy with deletion mutations amenable to exon 53 skipping. The efficacy and safety results were based on interim analyses at week [...]

Italfarmaco: Topline Data from Phase 2 Trial with Givinostat

July 2, 2021

Italfarmaco has announced results for their Givinostat trial in Becker muscle dystrophy (BMD). Although the change in total fibrosis in muscle biopsy did not show a significant difference in 51 adult males with BMD, it did show a significant difference from placebo on muscle MRI confirming the ability of Givinostat to counteract muscle deterioration. Italfarmaco [...]

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