Duchenne muscular dystrophy (DMD)
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...
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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...
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Mar 11, 2025
Neurotech’s ENCELTO Becomes First FDA-Approved Treatment for MacTel Type 2; Plus Therapeutics’ Rhenium Obisbemeda Gets FDA Orphan Status; Novo Nordisk Cuts WEGOVY to $499/Month; Capricor’s Deramiocel Wins FDA Priority Review; Celltrion’s OMLYCLO Becomes First Interchangeable XOLAIR Biosimilar
Neurotech’s ENCELTO Becomes First FDA-Approved Treatment for Macular Telangiectasia Type 2 Neurotech Pharmaceuticals, Inc. has announced that the FDA has approved ENCELTO (revakinagene taroretcel-lwey) for the treatment of Macular Telangiectasia type 2 (MacTel), a progressive neurodegenerative retinal disease th...
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Jan 07, 2025
Chimerix Submits Dordaviprone NDA for Accelerated Approval in Recurrent H3 K27M-Mutant Diffuse Glioma to FDA; NMD Pharma’s NMD670 Receives FDA Orphan Drug Designation for Charcot-Marie-Tooth Disease; Verastem’s Avutometinib NDA Accepted for KRAS Mutant Ovarian Cancer; Capricor Submits BLA for Deramiocel in Duchenne Muscular Dystrophy; Axsome Completes Phase III Trial of AXS-05 for Alzheimer’s Agitation
Chimerix Submits Dordaviprone NDA for Accelerated Approval to FDA for Recurrent H3 K27M-Mutant Diffuse Glioma Chimerix announced the submission of a NDA to the FDA for dordaviprone. The application seeks accelerated approval for the treatment of recurrent H3 K27M-mutant diffuse glioma, a rare and aggressive brai...
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Aug 20, 2024
In Search for a Curative Treatment Option for Duchenne Muscular Dystrophy
Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has recently achieved a significant milestone with the FDA's approval of AMONDYS 45 (casimersen). This antisense oligonucleotide, developed from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, is now approved for treati...
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Aug 20, 2024
Is the Cure for Duchenne Muscular Dystrophy in the Pipeline?
A rare muscle disorder, Duchenne Muscular Dystrophy (DMD), approximately affects 1 in 3,500 male births globally. In 2023, the total number of prevalent cases of Duchenne Muscular Dystrophy in the 7MM (The US, EU5, and Japan) was around 31,400. The United States encompasses the highest prevalent population of Duche...
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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...
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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...
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Oct 24, 2016
Sarepta’s Unusual Journey
Before approving an investigational drug, safety and effectivity needs to be proven. Something different happened with Sarepta’s Muscular dystrophy drug Eteplirsen, as its luck changed completely when the U.S. Food and Drug Administration on 19th September 2016 approved Sarepta Therapeutics' Exondys 51 (eteplirsen) ...
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Dec 22, 2014
DelveInsight’s Genetic disorders based Gene Therapy Reports
Gene Therapy to “fix” Genetic disorders! "We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes, "quotes James Watson. This fate and our future is locked in our genes, and Gene therapy is unlocking these doors. Many physicians are predicting that in twent...
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