Duchenne muscular dystrophy

Nov 01, 2021

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...

Oct 05, 2021

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...

Dec 17, 2019

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...

Dec 05, 2019

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...

Aug 20, 2019

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-...

Sep 11, 2018

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...

Sep 23, 2016

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...