Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disorder that affects muscle strength and movement due to the loss of motor neurons in the spinal cord. It has several subtypes, ranging from severe forms that appear in infancy (Type 1) to milder forms with later onset and slower progression (Types 2, 3, and 4). SMA can impact mobility, breathing, and daily activities, but advances in treatment, including gene and drug therapies, offer hope for improved outcomes. Support, specialized care, and early intervention are key to managing SMA and enhancing quality of life.
Maine Resources
MaineHealth - Neuromuscular Disorders - MaineHealth’s neurology team provides specialized care for neuromuscular disorders, including spinal muscular atrophy (SMA). They offer comprehensive diagnosis, treatment, and management options to help patients maintain mobility and quality of life. Services include advanced testing, rehabilitation, and access to new therapies.
Maine Newborn Bloodspot Screening Program - Identifies rare but serious conditions, including Spinal Muscular Atrophy (SMA), in newborns. Early detection through this statewide screening allows for timely medical intervention, improving outcomes for affected infants. The program ensures families receive support, follow-up care, and access to treatment options when necessary.
Northern Light Health - Pediatric Neurology - Northern Light Health’s Pediatric Neurology program provides specialized care for children with neurological and neuromuscular conditions, including other disorders affecting the brain and nervous system. Their team offers diagnostic evaluations, treatment plans, and ongoing management to improve quality of life for young patients and their families.
Regional Resources
Boston Children's Hospital - Offers advanced diagnostics, treatment options, and multidisciplinary support for children with Spinal Muscular Atrophy (SMA). Their approach includes genetic testing, innovative therapies like gene and drug treatments, and specialized rehabilitation to improve mobility and quality of life. The hospital’s team works closely with families to create individualized care plans.
Massachusetts General Hospital – The Adult SMA Clinic at Mass General provides specialized, team-based care for individuals with Spinal Muscular Atrophy (SMA). Their approach integrates neurology, pulmonology, physical therapy, nutrition, and other specialties to create comprehensive, personalized treatment plans. The clinic focuses on early intervention, advanced therapies, and long-term management to improve mobility, breathing, and overall quality of life for patients with SMA.
National Resources
Cure SMA - Dedicated to supporting individuals and families affected by spinal muscular atrophy (SMA) through research, advocacy, and community programs. The organization funds critical research to develop treatments and improve care while also providing resources, education, and support services to those living with SMA. Cure SMA advocates for policies that enhance quality of life and accelerate medical advancements.
Gwendolyn Strong Foundation (GSF) - This organization has branded themselves with the phrase, “Never Give Up” as a way to inspire courage among people in their community. GSF is committed to supporting individuals and families affected by Spinal Muscular Atrophy (SMA) through advocacy, awareness, and community engagement. Inspired by the resilience of those living with SMA, the organization raises funds for research, provides educational resources, and fosters a supportive network. Additionally, they offer bi-annual micro grants ranging from $250 to $2,500 to help individuals with SMA access essential resources and support. Their mission is to empower and uplift those facing SMA while working toward a future with better treatments and improved quality of life.
Miracle for Madison & Friends - Dedicated to supporting research, awareness, and assistance for individuals with spinal muscular atrophy (SMA). Inspired by Madison, a child living with SMA, the organization raises funds to advance treatments, provide resources for affected families, and promote community involvement. Through events, fundraising efforts, and advocacy, they strive to improve the lives of those with SMA while working toward a cure.
Muscular Dystrophy Association (MDA) - Committed to transforming the lives of individuals affected by neuromuscular diseases, including spinal muscular atrophy (SMA). MDA funds groundbreaking research, provides medical and community support services, and advocates for access to care and treatments. Through specialized clinics, summer camps, and educational resources, MDA empowers individuals and families living with SMA and other neuromuscular conditions.
National Institute of Neurological Disorders and Stroke (NINDS) - Provides an overview of Spinal Muscular Atrophy (SMA), a genetic disorder that affects nerve cells in the spinal cord, leading to progressive muscle weakness and loss of movement. NINDS supports research into the causes, treatments, and potential cures for SMA while also offering educational resources for patients and caregivers.
National Organization of Rare Diseases (NORD) - Details the different subtypes of SMA, ranging from severe infantile-onset forms to milder adult-onset types. NORD is dedicated to improving the lives of people with rare diseases through advocacy, education, research, and patient support.
SMA Angels Charity - Dedicated to supporting individuals and families affected by Spinal Muscular Atrophy (SMA). The organization focuses on raising awareness, funding research for treatments and a cure, and providing financial assistance to families in need. Through advocacy, grants, and community support, SMA Angels strives to improve the quality of life for those living with SMA.