Duchenne Muscular Dystrophy Treatment
Aug 05, 2025
Anbogen’s ABT-301 Cleared by FDA for Phase I/II Colorectal Cancer Trial; Dyne’s DYNE-251 Gets FDA Breakthrough Tag for Duchenne Muscular Dystrophy; BMS’s BREYANZI Accepted for FDA Priority Review in MZL; Clarametyx’s CMTX-101 Earns FDA Fast Track and QIDP for Infections; Anixa Begins FDA-Approved IND Transfer for Phase II Breast Cancer Vaccine Trial
Anbogen Secures FDA Nod to Begin Phase I/II Trial of ABT-301 Triplet in Advanced Colorectal Cancer Anbogen Therapeutics has received FDA clearance to initiate a Phase I/II clinical trial of ABT-301 in combination with tislelizumab and bevacizumab for patients with metastatic colorectal cancer (mCRC). The study w...
Read More...
Jul 22, 2025
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths; ImCheck’s ICT01 Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia; RedHill Biopharma Gets Positive FDA Feedback on RHB-204 for Crohn’s Disease Approval Pathway; FDA Grants Fast Track Designation to ProMIS Neurosciences’ PMN310 for Alzheimer’s Disease; Oruka Therapeutics Receives IND Clearance for Phase IIa Trial of ORKA-001 in Psoriasis
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths Sarepta Therapeutics, Inc. has voluntarily and temporarily paused all shipments of ELEVIDYS (delandistrogene moxeparvovec) in the U.S., effective July 22, 2025, following engagement with the FDA on safety labeling u...
Read More...
Aug 23, 2024
What Does the Future Hold for Gene Therapy in the Duchenne Muscular Dystrophy (DMD) Treatment Market?
Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Gene therapy for Duchenne Muscular Dystrophy (DMD) is poised to revolutionize the treatment landscape by addressing the underlying genetic cause of the...
Read More...
Jun 19, 2024
Gene therapy: Following Pfizer’s Unexpected Failure, Sarepta is celebrating its much-awaited full approval! Sarepta’s stock price has shot up over 32%
Duchenne muscular dystrophy (DMD) is a severe genetic disorder primarily affecting young boys between the ages of two and three. As per Delveinsight’s estimates, there were approximately 16K DMD patients in 2023 in the United States, with ˜75% of the patients below the age of 15. This condition leads to progressive...
Read More...
Jun 30, 2023
Sarepta’s ELEVIDYS: First Gene Therapy for Duchenne Muscular Dystrophy (DMD) Treatment
Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that affects 1 in every 3,500 to 5,000 male neonates worldwide. It is caused by mutations in the gene encoding the protein dystrophin. These genetic changes emerge as developmental delays and, in more severe types of DMD, limb weakness, loss of indep...
Read More...
Dec 24, 2019
Roche, Sarepta collaborates; FDA approval for Merck’s Ervebo, and Allergan’s Ubrelvy
Roche has entered into a licensing partnership with Sarepta Therapeutics worth USD 1.15 Billion. Roche has agreed to license in Sarepta’s gene therapy ‘SRP-9001’ for Duchenne muscular dystrophy (DMD), though which Roche will gain access to exclusive rights to it globally except in the US. As per t...
Read More...
-Agonist.png "Toll-like Receptors Agonist")
