Press Releases
STM-01 Enters Phase 2 Clinical Trial for Duchenne Muscular Dystrophy
Secretome Therapeutics has announced the start of THRIVE-2, a Phase 2 clinical study evaluating its investigational therapy STM-01 in people living with Duchenne muscular dystrophy (DMD). The study is now open for enrollment, with the first participant expected to enroll soon.What Is THRIVE-2?THRIVE-2 is designed as a multicenter, multi-dose,...
Research
Keros Therapeutics Doses First Patient in Phase 2 Rinvatercept Trial
Keros Therapeutics has announced an important step in the development of Rinvatercept, an investigational treatment for Duchenne muscular dystrophy (DMD). The company reported on September 28, 2026, that the first patient has been dosed in its Phase 2 clinical trial evaluating Rinvatercept in people with DMD.The company expects initial...
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Research
Tevard’s tRNA Therapy Targets Nonsense Mutations to Restore Full-Length Dystrophin in DMD
Tevard’s suppressor tRNA therapy could offer a new way to address nonsense mutations in...
Research
Beyond Micro-Dystrophin: Could Full-Length Dystrophin Be the Next Step in DMD Gene Therapy?
Renzhi Han, PhD, at Indiana University is advancing a new direction in gene therapy...
Research
tRNA Research for Nonsense Mutations Offers New Hope for Genetic Disorders
Researchers at the University of Toronto, University Health Network and SickKids have developed a...
Research
PBGENE-DMD Enters Clinical Trials: First Patient Dosed in Duchenne Gene-Editing Study
Precision BioSciences has reached an important milestone in Duchenne muscular dystrophy (DMD) gene editing....
Research
HLB Panagene Turns to PNA-AOC as a New Approach for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) remains a major challenge for drug developers because many therapeutic...
Research
Sarcomatrix Plans First Human Trial of S-969 for Duchenne Muscular Dystrophy in 2027
For families affected by Duchenne muscular dystrophy (DMD), every new clinical development offers hope....
Research
DYNE-251 Phase 3 FORZETTO Clinical Trial of Z-Rostudirsen Officially Begins for DMD
Dyne Therapeutics has announced the start of the Dyne Therapeutics Phase 3 FORZETTO Trial...
Research
Del-Zota SAFARI44 Phase 3 Trial: A New Exon 44 Skipping Study for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) research continues to move forward with the launch of the...
Research
BioMarin Launches BMN 351 Exon 51 Skipping Therapy Phase 2 Clinical Trial for Duchenne Muscular Dystrophy
BioMarin Pharmaceutical has officially launched a new Phase 2 extension study (NCT07573631) evaluating BMN...
Research
Precision BioSciences Announced the Start of Phase 1/2a (FUNCTION-DMD) Study of PBGENE-DMD in Ambulatory Patients with Duchenne
Precision BioSciences announced the commencement of a Phase 1/2a trial of PBGENE-DMD (NCT07429240) in...
Research
ELEVIDYS ENHANCE Study: A Phase 4 Trial Begins to Evaluate Safety and Effectiveness in Duchenne Muscular Dystrophy
The ENHANCE study is a Phase 4 clinical trial designed to evaluate the real-world...
Research
Medera’s Gene Therapy for DMD Cardiomyopathy Gains Momentum with FDA Fast Track Designation
A promising advancement in gene therapy for DMD cardiomyopathy has emerged as Medera Inc....
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Press Releases
Satellos Completes Enrollment in Phase 2 Forazapadin Trial for Duchenne Muscular Dystrophy
Satellos Bioscience has announced the completion of enrollment in BASECAMP, a Phase 2 clinical...
Press Releases
STM-01 Enters Phase 2 Clinical Trial for Duchenne Muscular Dystrophy
Secretome Therapeutics has announced the start of THRIVE-2, a Phase 2 clinical study evaluating...
Care Guidelines
Duchenne Muscular Dystrophy Speech Delay: Understanding Language Development in Children with DMD
Duchenne muscular dystrophy speech delay is an important developmental concern that families and healthcare...
Research
Keros Therapeutics Doses First Patient in Phase 2 Rinvatercept Trial
Keros Therapeutics has announced an important step in the development of Rinvatercept, an investigational...
Editorials
The Systemic Failure of DMD Associations: Why Duchenne Patients Are Still Waiting
Duchenne muscular dystrophy (DMD) is not a newly discovered condition, nor is it an...
Editorials
In Which Countries Is Elevidys Approved? Is Elevidys Approved In Europe?
Elevidys (delandistrogene moxeparvovec) is a gene therapy developed for Duchenne muscular dystrophy (DMD). It...
Press Releases
Exegenesis Bio and Modalis Collaborate to Advance MDL-201 for Duchenne Muscular Dystrophy
Exegenesis Bio and Modalis Therapeutics Corporation have announced a research collaboration and license agreement...
Research
Tevard’s tRNA Therapy Targets Nonsense Mutations to Restore Full-Length Dystrophin in DMD
Tevard’s suppressor tRNA therapy could offer a new way to address nonsense mutations in...