FAQs about SMA type 0
Type 0 is the rarest form of spinal muscular atrophy (SMA) — only a few dozen cases have been documented, in part because this form of the disease was only first described in the late 1990s. It’s likely that many babies with SMA type 0 die before they get a formal diagnosis of the disease. Consequently, the exact prevalence of SMA type 0 is not known.
There is no cure for any form of spinal muscular atrophy (SMA). Three disease-modifying treatments that can slow SMA’s progression are currently approved, but there is minimal published data on the use of any of these therapies in babies with type 0 disease. A few reports have shown mild benefits for SMA type 0 babies given disease-modifying treatment, though treated children continued to experience substantial symptoms.
Babies with spinal muscular atrophy (SMA) type 0 usually have substantial breathing difficulties when they are born. A ventilator can help these patients breathe.
Babies born with spinal muscular atrophy (SMA) type 0 do not survive until adulthood. They have substantial symptoms at birth, and they generally do not survive past the first few months of life.
Babies born with spinal muscular atrophy (SMA) type 0 typically do not live more than a few months.