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These little girls were born with the same cruel genetic illness... but baby Amelia will never suffer like big sister Maisie - thanks to a miracle £75,000-a-dose jab

Portia Thorman of the Spinal Muscular Atrophy charity SMA, said these new treatments were not available when her son Ezra, pictured left, was born.

'It's a real-life Hunger Games': a lifesaving drug costs $2m, but not every child can get it | Pharmaceuticals industry

Zolgensma is a revolutionary gene therapy that can stop a deadly childhood condition called SMA in its tracks. It’s also one of the most expensive drugs in the world

NICE recommends risdiplam for spinal muscular atrophy

NICE has published draft guidance recommending risdiplam (Evrysdi) as part of a managed access agreement (MAA) for the treatment of the rare genetic disorder spinal muscular atrophy (SMA). - News - PharmaTimes

UK's NICE recommends Roche's Evrysdi

Oral drug for spinal muscular atrophy to be available on NHS in England