Key details Disabling condition: Duchenne muscular dystrophy (DMD) is a severe, genetic condition that causes progressive muscle weakness due to the absence of dystrophin, a protein essential for maintaining muscle integrity. Increasing scientific interest in whether...
Key details Current treatment limitations: While ischaemic strokes – which account for nearly 90% of all strokes – are currently treated with clot removal and supportive rehabilitation, there remains a significant unmet need for therapies that directly address...
Key details New trial approval: The South Korean Ministry of Food and Drug Safety has approved a Phase 2a trial for a new umbilical cord stem cell therapy to treat Charcot-Marie-Tooth disease (CMT), one of the most commonly inherited nerve diseases.[1] Early clinical...
Key details Understanding Lesch-Nyhan syndrome: This rare, recessive condition linked to the X chromosome primarily affects boys, and is characterised by a deficiency of the HPRT enzyme, leading to severe motor dysfunction, uric acid overproduction, and compulsive...
A six-year-old girl named Zara has become the first child in Australia to receive an infusion of her own stored cord blood as treatment for cerebral palsy outside of a clinical trial. Experts hope that this milestone will pave the way towards broader availability,...
Researchers are investigating a novel placenta-derived stem cell therapy for multiple sclerosis, a currently incurable degenerative neurological condition. A recent phase 1 clinical trial, with findings published in Scientific Reports, assessed the safety and...