Key points from article :
Gene therapy Zolgensma offers hope to infants with a type of severe spinal muscular atrophy (SMA).
Around 80 babies and young children with type 1 SMA could benefit from the treatment each year in England.
Described in the British Medical Journal as "the most expensive drug course of treatment ever".
Although the drug was hugely expensive, its use is justified by the exceptional impact.
If the drug is given to children in their first six months of life, it might come close to being a cure by halting disease progression.
The drug contains a replica of the missing gene SMN1.
It helped babies breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment.
Only limited data about the impact of Zolgensma is available from clinical trials.
"For some babies who are diagnosed before they have symptoms, it might come close to being a cure," - Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at Nice.
