The Emirates Drug Establishment (EDE) has announced the approval of Itvisma (onasemnogene abeparvovec), a gene therapy that uses an adeno-associated viral vector to treat spinal muscular atrophy (SMA) in eligible adults and children aged two years and above.
According to a new study, some persons with motor neuron disease (MND) and frontotemporal dementia (FTD) share the same rare genetic diseases that cause other neurodegenerative illnesses.
Stanford (California) [US], January 19 (ANI): A new ultra-rapid genome sequencing approach developed by Stanford Medicine scientists and their collaborators was used to diagnose rare genetic diseases in an average of eight hours -- a feat that's nearly unheard of in standard clinical care.
New Delhi [India], Dec 13, (ANI): The Indian Council of Medical Research (ICMR) on Friday released the draft guidelines for ensuring that rare genetic therapies and clinical trials can be performed in an ethical, scientific and safe manner in India.