Babies at MFT have been part of groundbreaking research which found that a single dose of a treatment for a respiratory infection in infants can help cut hospital admissions by more than 80 per cent.
The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.
ITV Tyne Tees and Granada Reports recently featured the journey of two-year-old Teddi Shaw, who became the first child in the UK to receive NHS life-saving gene therapy treatment, Libmeldy® at MFT.
For a nine-year old boy and his family, being part of Duchenne Muscular Dystrophy clinical research at NIHR Manchester CRF at Royal Manchester Children's Hospital has been ‘amazing,’ for many reasons.
Pioneering research has shown dramatic results for treating children with high-risk leukaemia (a type of blood cancer), who previously had no hope of recovery after all other treatment options failed.
Researchers tell an international conference that a new gene therapy for Sanfilippo disease – also known as childhood dementia – has shown promising early results in a proof-of-concept study.