News & Updates | Duchenne Muscular Dystrophy

News & Resources

Dyne Therapeutics: FDA Clinical Hold on IND Application for DYNE-251

January 20, 2022

As part of our commitment to the Duchenne muscular dystrophy community to deliver the most up-to-date information, Jesse’s Journey is providing you with the following news regarding Dyne Therapeutics. The U.S. Food and Drug Administration (FDA) announced a clinical hold on the Dyne Therapeutics New Drug (IND) application for DYNE-251 - their exon 51 skipping [...]

SOLID Biosciences: Letter to the Duchenne Community

January 13, 2022

Jesse’s Journey is pleased to share an update regarding the IGNITE-DMD Phase I/II clinical trial of SGT-001 from SOLID Biosciences Inc., as well as the progress in expanding their pipeline of treatments for Duchenne muscular dystrophy. Earlier this week SOLID provided an update on its 2022 strategic priorities and other business initiatives at the Annual [...]

Sarepta Therapeutics: Update on Gene Therapy SRP-9001 & SRP-9001-102

January 13, 2022

Sarepta Therapeutics recently announced topline results from Part 2 of Study SRP-9001-102 (Study 102) that showed statistically significant functional improvements for the treatment of Duchenne muscular dystrophy: “We are delighted to report positive results for Part 2 of our blinded, placebo-controlled Study 102 in Duchenne, where the 48-week functional benefits of SRP-9001 in patients dosed [...]

REGENXBIO: FDA Clearance of IND for Clinical Trial of RGX-202

January 6, 2022

Jesse’s Journey is pleased to share the news that REGENXBIO has received clearance from the U.S. Food and Drug Administration (FDA) on their investigational gene therapy drug, RGX-2022. This means that they can move forward with the initiation of their first clinical trial with ambulatory boys aged 4-11 years with Duchenne muscular dystrophy. They hope [...]

Edgewise Therapeutics: Positive Topline Results From the EDG-5506 Phase 1b Clinical Trial in Adults With Becker Muscular Dystrophy (BMD)

January 5, 2022

Jesse’s Journey is pleased to share the news that Edgewise Therapeutics has announced positive topline results from the EDG-5506 Phase 1b clinical trial in adults with Becker muscular dystrophy (BMD). “The completion of this clinical trial in BMD adults is an important milestone for Edgewise. We’re excited as we advance into Phase 2 clinical trials [...]

Dystrogen Therapeutics: First Human Dosing of Novel Chimeric Cell Therapy for Duchenne Muscular Dystrophy and Reports 6-week Clinical Outcomes

January 4, 2022

Jesse’s Journey is pleased to share the news that Dystrogen Therapeutics Corporation, a clinical-stage company developing a dystrophin replacement approach using chimeric cell therapy, announced the dosing of the first patient in their Phase 1 human pilot clinical study of DT-DEC01, Dystrophin Expressing Chimeric cells, or DEC, for the treatment of Duchenne muscular dystrophy (DMD). [...]

Pfizer: Update on Phase 1B Open-Label Mini-Dystrophin Gene Therapy Trial

December 20, 2021

Over the holidays we received some tragic news. We are deeply saddened to learn that a young man participating in the non-ambulatory cohort of Pfizer’s Phase 1b mini-dystrophin gene therapy trial for Duchenne muscular dystrophy has passed away. It is very difficult to find the right words to express how deeply this has touched our [...]

Dyne Therapeutics: IND Application to Initiate Clinical Trial of DYNE-251

December 6, 2021

Jesse’s Journey is pleased to share the latest news from Dyne Therapeutics. Dyne Therapeutics is a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. Dyne has announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a [...]

Santhera and ReveraGen: Positive Topline Results with Vamorolone (Completion of the VISION-DMD Study at 48 Weeks)

November 30, 2021

Jesse’s Journey is pleased to provide the latest update from Santhera Pharmaceuticals and ReveraGen BioPharma, Inc. regarding the completion of the 48-week VISION-DMD study: The 48-week top-line data readout for vamorolone shows that the statistically robust efficacy demonstrated at 24-weeks is maintained across multiple efficacy endpoints at 48 weeks. In addition, vamorolone treatment at both [...]

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 [...]

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