Duchenne muscular dystrophy

Bristol-Myers Squibb’s Opdivo and Yervoy Combo Fails in Phase III Trial Bristol-Myers Squibb has reported that their Opdivo and Yervoy checkpoint inhibitor combo failed a phase III trial as adjuvant (post-surgery) therapy for renal cell carcinoma (RCC), the most frequent type of kidney cancer. The CheckMate -914...

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. It is usually observed between the ages of three and six. Duchenne Muscular Dystrophy signs and symptoms include pelvic muscles atrophy, followed by in...

Progress is driven by innovation. When it comes to developing novel medications and therapeutic biological products, the FDA's Center for Drug Evaluation and Research (CDER) assists the pharmaceutical sector at every stage of the process. CDER offers scientific and regulatory assistance needed to bring innovative m...

Pfizer tightens DMD patient criteria after serious adverse events in phase 3 gene therapy trial Pfizer is tightening up the criteria for Duchenne muscular dystrophy patients to partake in phase 3 clinical trials of its experimental gene therapy after three severe events were cropping up in ongoing studies. Th...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder usually diagnosed in young boys, gradually weakening muscles across the body until the heart or lungs fail. Symptoms often show up by the age of 5; as the disease progresses, patients tend to lose the ability to walk around the age of 12. As per a re...

Wave Life Sciences has recently announced the discontinuation of its drug suvodirsen, for the treatment of a rare Duchenne Muscular Dystrophy mutation.  Duchenne Muscular Dystrophy is a progressive worsening of the skeletal and heart muscles resulting in weakness. The disorder, is due to deficient prot...

Bristol-Meyers Squibb and Acceleron Pharma have announced that US FDA committee will review BMS’s supplemental Biologics License Application (sBLA) for its Reblozyl in MDS. Reblozyl ((luspatercept-aamt) has recently been approved by the regulatory authority to cure a rare type of blood disorder, Beta-thalassemi...

Juvenescence closes a USD 100 Million Series B round Juvenescence, a life sciences company focused on treating ageing problems, has successfully raised USD 100 Million in a Series B round. The investors who took part in the funding were Grok Ventures, the investment company of Mike Cannon-Brookes (Atlassian co-...

Nucala of GlaxoSmithKline exhibits splendid benefits to other IL-5 Inhibitors in Asthma, however flunks as a COPD Drug A Complete Response Letter is received by GSK from the U.S. Food and Drug Administration (FDA) for Nucala (mepolizumab) as COPD treatment. Nucala, which targets the IL-5 pathway, was initially appro...

The Food and Drug Administration approved the recent controversial drug to treat Duchenne muscular Dystrophy, which is a rare disease that confines boys to wheelchairs and condemns them to an early death. The decision was made after months of debate between the Agency and Sarepta Therapeutics, regarding the evidence...