Low myostatin levels linked to lack of success of anti-myostatin drugs for muscle-wasting conditions
30 Nov 2017, 2:04 p.m.
Levels of myostatin, a protein that prevents muscle growth, could influence how well the body responds to anti-myostatin treatments for muscle-wasting conditions such as Duchenne Muscular Dystrophy (DMD). This breakthrough could help predict which patient
A state-of-the-art stem cell research facility is now open at Great Ormond Street Hospital (GOSH), increasing our capacity for cutting edge research into regenerative medicine.
Advances in the treatment of the severe immune disorder Wiskott-Aldrich syndrome
20 Nov 2017, 2:29 p.m.
Researchers at Great Ormond Street Hospital (GOSH) have made an important breakthrough in understanding how the immune system becomes disrupted in Wiskott-Aldrich syndrome (WAS), a genetic disorder where the patient’s immune cells begin attacking their ow
Drug for Duchenne Muscular Dystrophy shows promise in clinical trial
21 Aug 2017, 12:02 p.m.
An international clinical trial has found that the drug ataluren can improve mobility for children with a severe form of Duchenne muscular dystrophy (DMD).
£75,000 boost for Duchenne Muscular Dystrophy gene therapy research
8 Aug 2017, 4:03 p.m.
GOSH Biomedical Research Centre researcher Dr John Counsell has been awarded up to £75,000 of funding from the UCL Therapeutic Acceleration Support (TAS) Fund for a study exploring the use of novel vectors in gene therapy for Duchenne Muscular Dystrophy (