FAQs about SMA-LED

Category: SMA-LED

Spinal muscular atrophy with lower extremity predominance (SMA-LED) is a rare type of spinal muscular atrophy, with symptoms usually starting in infancy, and marked by weakness in the lower limbs.

Category: SMA-LED

Yes. Due to the difficulty in diagnosing spinal muscular atrophy with lower extremity predominance (SMA-LED), some people may receive a diagnosis as late as adulthood. In fact, about one-quarter of those with SMA-LED may not develop symptoms, such as muscle weakness, until they are adults.

Category: SMA-LED

Children with spinal muscular atrophy with lower extremity predominance (SMA-LED) may have delayed motor development, and some may never walk. Others may have a waddling or unsteady gait, and some may require specialized equipment.

Category: SMA-LED

There is no cure for SMA-LED at this point in time. Available treatments can help ease the disease symptoms and contribute to a better quality of life.

Category: SMA-LED

At this point, there is no gene therapy for SMA-LED, which is caused by mutations in the DYNC1H1 or BICD2 genes.