The family of a girl who campaigned for a drug to treat her muscle-wasting disease to be made available on the NHS said they understand she is not eligible to receive it.
Heidi Prescott-Both, 11, has spinal muscular atrophy (SMA).
She was vocal in calls for Spinraza to be made available to treat the condition, but is now “devastated” she does not qualify to receive it.
NICE said experts found the eligibility criteria “broadly appropriate”.
SMA is a genetic condition which affects the nerves in the spinal cord, weakening muscles and causing problems with movement, breathing and swallowing.
Heidi, from Wolverhampton, was diagnosed at age three.
She struggles to walk and will be having surgery on a spinal curve in around six months time.
While not a cure, trials have shown that Spinraza, the trade name of drug nusinersen, can slow the effects of SMA in some cases.
Her mother, Katie Prescott, said the family were “over the moon” when it was approved for use.
But NICE are only allowing nusinersen to be used by patients who it believes would help with its research into the drug through a “managed access arrangement”.
It said its independent appraisal committee “acknowledged the need to manage risks associated with the identified uncertainties”.
And after viewing a list of criteria of cases which are eligible, Mrs Prescott believes Heidi is among 15% of patients who will not qualify to receive it.
Mrs Prescott said: “Heidi is heartbroken.
“Fingers crossed something will happen because she deserves treatment as much as everyone else.”
A statement from NICE said: “Patient and clinical experts considered that the eligibility criteria were broadly appropriate.”
The charity TreatSMA said it has lodged an official appeal with NICE, claiming there is “no medical evidence” Spinraza is not effective within the excluded population.
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