Duchenne muscular dystrophy

Neuromuscular disorders (NMD) encompass a broad spectrum of conditions impacting the peripheral nervous system, which comprises all motor and sensory nerves linking the brain and spinal cord to the body. These disorders comprise various classes of diseases, such as muscular dystrophies, myopathies, motor neuron dis...

Merck and Moderna Initiate INTerpath-002, a Phase III Study Evaluating V940 in Combination with KEYTRUDA for Adjuvant Treatment of Patients with Certain Types of Resected NSCLC Merck (also known as MSD outside the United States and Canada) and Moderna, Inc. have commenced the INTerpath-002 trial—a crucial Phase ...

Eylea HD Injection 8 Mg Approved By FDA for Treatment of Wet AMD, DME, and Diabetic Retinopathy The FDA has approved Regeneron Pharmaceuticals’ EYLEA HD (aflibercept) Injection of 8 mg for the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retin...

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...

FDA Approves Jardiance for the Treatment of Type 2 Diabetes in Children 10 Years and Older Boehringer Ingelheim and Eli Lilly and Company announced that the FDA has approved Jardiance® (empagliflozin) 10 mg and 25 mg tablets to decrease blood sugar together with diet and exercise in children 10 years and older w...

FDA Grants Fast Track Status to KYV-101 for Refractory Lupus Nephritis Treatment Kyverna Therapeutics announced that the FDA has given Fast Track status to KYV-101, a treatment for patients suffering from resistant lupus nephritis (LN). KYV-101 is an innovative therapy that uses anti-CD19 chimeric antigen recept...

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy Sarepta Therapeutics, Inc., a pioneer in precision genetic medicine for rare diseases, announced that the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in favor of...

Actinium Announces Positive Top-line Results from Pivotal Phase III SIERRA Trial of Iomab-B Actinium Pharma is on track to submit its targeted radiotherapy for AML patients requiring a bone marrow transplant in the United States, boosted by top-line data from a pivotal trial. The SIERRA trial of Iomab-B, an anti...

Zealand Pharma Announces the Positive Topline Results from its Phase III trial of Glepaglutide Zealand Pharma A/S, a biotech company specializing in peptide-based medicines, announced positive topline results from its phase III trial of glepaglutide. In the evenly randomized double-blind trial, 106 patients with...