FAQs about Finkel type SMA
Finkel type spinal muscular atrophy (FSMA) is a form of adult-onset spinal muscular atrophy that is caused by mutations in the VAPB gene. It has a slow progression, and is marked by weakness in the lower limbs that gradually spreads to upper limbs.
Yes, only adults are known to have Finkel type spinal muscular atrophy (FSMA).
Yes, due to the slow progression of Finkel type SMA (FSMA), the majority of people are able to walk. Later in life, adaptive equipment such as walkers or wheelchairs may be needed.
No, there is no cure for Finkel type SMA (FSMA) or spinal muscular atrophy in general. Treatment for FSMA is usually focused on the management of disease symptoms and on improving quality of life. The use of specialized equipment and physical therapy can be of benefit to this patient population.
At this point, there is no gene therapy suitable for Finkel type spinal muscular atrophy (FSMA). Children up to age 2 with one of the main types of SMA, however, can be eligible to receive treatment with Zolgensma — the only gene therapy approved for SMA.