This webinar, featuring an expert panel of speakers, aims to help healthcare professionals to understand what spinal muscular atrophy (SMA) is, how it presents, how to treat it, and why early detection, diagnosis and treatment are essential to children's outcomes.

NICE draft draft guidance does not recommend gene therapy OTL-200 for metachromatic leukodystrophy in children, but NICE says it would welcome further discussions between NHS England and the company making the treatment to resolve the uncertainties highlighted by the committee.

NICE has published final draft guidance recommending the treatment and final guidance is expected in July.

More people with the rare genetic disorder spinal muscular atrophy (SMA) are to benefit from the treatment, says NICE.

Author: Louise Newson, general practitioner, Birmingham. Reviewed by Luke Koupparis, general practitioner, Bristol.