A stem cell therapy for hypoplastic left heart syndrome (HLHS), a serious congenital heart defect that affects blood flow through the heart, has entered a key clinical trial stage. The therapy aims to strengthen the heart and improve outcomes for children with the...
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 recent study conducted at UMC Utrecht, Netherlands, has shown that mesenchymal stem cells (MSCs) are a safe treatment for brain damage caused by stroke in newborns, and may have a significant positive effect. Perinatal stroke and its consequences Perinatal stroke is...
A groundbreaking therapy based on transplanting mitochondria into blood stem cells has shown encouraging results in an ongoing, phase 2 trial. If successful, the therapy could not only treat rare, incurable diseases, but also potentially help reverse some of the...
