Connecticut Children’s Neuromuscular Program is program that is funded by the Muscular Dystrophy Association (MDA). This multidisciplinary team evaluates and treats children of all ages affected by neuromuscular conditions, including:
- Inherited and acquired neuropathies
- Muscular dystrophy (Duchenne and Becker; congenital)
- Metabolic diseases
- Motor neuron disorders
- Myoasthenia gravis
- Congenital myopathies
- Spinal Muscular Atrophy (SMA)
We provide the most advanced treatments and clinical trials for Duchenne muscular dystrophy and spinal muscular atrophy (SMA). In clinical trials we administered for Spinraza, which was approved by the FDA in December 2016, infants treated before showing symptoms gained more motor milestones than those treated after showing symptoms. In 2018, we received a five-year extension of work with Spinraza from drugmaker Biogen and received a grant from Cure SMA to create a patient registry to follow patients over a number of years. A typical SMA patient will be able to achieve close to normal developmental milestones when treated early.
Integrated Clinical Care Team
Led by Gyula Acsadi, MD, PhD, the MDA Care Center is staffed by a highly skilled clinical team, including neurologists, a physiatrist, a nurse, physical therapists, occupational therapists and a nutritionist. The team collaborates with Connecticut Children’s orthopedic specialists, cardiologists and pulmonologists.