What is Spinal Muscular Atrophy?
Spinal Muscular Atrophy is treated with 3 medications in our database, including Zolgensma, Spinraza, EVRYSDI. 3 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Novartis, Biogen, Genentech.
Also known as:
- Inheritance
- Autosomal recessive
- Passed on when both parents carry the same gene change; often skips generations
- Age of Onset
- Variable
- Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsCharles University, Czech Republic — NA
Teachers College, Columbia University — NA
Gemma Biotherapeutics — PHASE1, PHASE2
Institut de Myologie, France — NA
Data is compiled from FDA regulatory filings and ClinicalTrials.gov, then processed through automated extraction; event classifications and dates may occasionally be misclassified. Verify against the linked FDA filing or trial record before clinical decisions. Updated periodically.
Treatments
3 FDA-approvedSource: openFDA + DailyMed · NDA / BLA labels with structured indications · refreshed weekly
FDA-approved (3)
Zolgensma
1 INDICATIONS AND USAGE ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (…
1 INDICATIONS AND USAGE ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated [see Adverse Reactions ( 6.2 )] . The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated [see Clinical Studies ( 14 )] . ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 ( SMN1) gene. ( 1 ) Limitations of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated. ( 1 , 6.2 ) The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated. ( 1 , 14 )
Spinraza
1 INDICATIONS AND USAGE SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligon…
1 INDICATIONS AND USAGE SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients ( 1 )
EVRYSDI
1 INDICATIONS AND USAGE EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. EVRYSDI is a survival of motor neuron 2 (SMN2) splicing modifier indica…
1 INDICATIONS AND USAGE EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. EVRYSDI is a survival of motor neuron 2 (SMN2) splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. ( 1 )
Source: ClinicalTrials.gov · synced daily · phases, status, and PI names normalized at ingest
Source: NPI Registry + PubMed · trial PI roles cross-referenced with ClinicalTrials.gov · NORD partners highlighted · ordered by verified-leader / active-researcher / listed-specialist tier
Rare Disease Specialist
Columbia University
Rare Disease Specialist
Newcastle University
Rare Disease Specialist
Istituto Gaslini
Rare Disease Specialist
Hospital Albert Einstein
Rare Disease Specialist
Hôpital Necker-Enfants Malades
Rare Disease Specialist
University of Liege
Pediatric Neurology / SMA Gene Therapy
Nationwide Children's Hospital / Ohio State University
Specialty unconfirmed
Hasselt University
Rare Disease Specialist
Kaohsiung Medical University Chung-Ho Memorial Hospital
Rare Disease Specialist
Kamila Řasová
Rare Disease Specialist
Alberta Children's Hospital
Rare Disease Specialist
Pediatric Rehabilitation Service - L'Escale Mother and Child Hospital
Rare Disease Specialist
Centre Hospitalier Régional de la Citadelle
Rare Disease Specialist
Barrow Neurological Institute
Rare Disease Specialist
Peking University Third Hospital
Rare Disease Specialist
MedStar Health Research Institute
Rare Disease Specialist
Centre d'Exploration et d'Évaluation Neuromusculaire
Rare Disease Specialist
RTI International
Rare Disease Specialist
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rare Disease Specialist
CHU Rouen
Rare Disease Specialist
Stanford University Medical Center
Pediatric Neurology / SMA
Children's Hospital of Eastern Ontario (CHEO)
Neurology / Neuromuscular Disorders
Fondazione IRCCS Istituto Neurologico Carlo Besta
Pediatric Neurology / SMA Clinical Trials
Nationwide Children's Hospital
Pediatric Neurology / Spinal Muscular Atrophy
Johns Hopkins University School of Medicine
Treatment Centers
8 centersSource: NORD Rare Disease Centers + NIH Undiagnosed Diseases Network (UDN) · centers verified active within last 12 months
Children's Hospital Colorado
📍 Aurora, Colorado
👤 Zachary Grinspan, MD
Arkansas Children's Hospital
📍 Little Rock, Arkansas
Boston Children's Hospital
📍 Boston, Massachusetts
Massachusetts General Hospital
📍 Boston, Massachusetts
👤 Matthew Frigault, MD
👤 Janssen Research & Development, LLC Clinical Trial
Children's Hospital of Orange County
📍 Orange, California
👤 Richard Neibeger, MD
Children's Hospital of Philadelphia
📍 Philadelphia, Pennsylvania
👤 Study Director
👤 Richard Neibeger, MD
Ann & Robert H. Lurie Children's Hospital of Chicago
📍 Chicago, Illinois
University of Arkansas for Medical Sciences
📍 Little Rock, Arkansas
Financial Resources
10 resourcesSource: manufacturer patient-assistance programs (PAP) + copay-card programs · NORD Patient Assistance · HealthWell Foundation + disease-specific foundation grants · links verified by automated cron
Good Days — Spinal Muscular Atrophy
Good Days
Spinal Muscular Atrophy
Patient Services Inc — Spinal Muscular Atrophy
Patient Services Inc
Spinal Muscular Atrophy
NORD Patient Assistance — Spinal Muscular Atrophy
NORD Patient Assistance
Spinal Muscular Atrophy
Patient Advocate Foundation — Spinal Muscular Atrophy
Patient Advocate Foundation
Spinal Muscular Atrophy
HealthWell Foundation — Spinal Muscular Atrophy
HealthWell Foundation
Spinal Muscular Atrophy
The Assistance Fund — Spinal Muscular Atrophy
The Assistance Fund
Spinal Muscular Atrophy
PAN Foundation — Spinal Muscular Atrophy
PAN Foundation
Spinal Muscular Atrophy
8 travel grants are also available for Spinal Muscular Atrophy patients — see Travel Grants below ↓
Travel Grants
8 grantsHelp Hope Live Spinal Muscular Atrophy Financial Assistance
Eligibility requires individuals to have a medically verified need related to Spinal Muscular Atrophy to qualify for community-based fundraising support. Participants must submit an application through the organization to be paired with a coordinator who assists in raising tax-deductible funds for out-of-pocket medical expenses.
Spinal Muscular Atrophy Copay Grant
PAN Foundation
Patients must have a confirmed diagnosis of spinal muscular atrophy and possess valid health insurance for their prescribed medications. Applicants are required to provide household income information and can apply online or by phone when the fund is open.
Novartis Patient Assistance Foundation, Inc. (NPAF)
Novartis Patient Assistance Foundation, Inc.
Assistance is primarily available to patients diagnosed with Myelodysplastic Syndromes (MDS) and their caregivers for costs related to treatment, travel, and lodging. Specific eligibility criteria, including financial need and insurance status, vary significantly across the individual programs listed.
Novartis Patient Assistance Program
Novartis
Eligibility is generally restricted to organ and tissue transplant candidates, recipients, and living donors who demonstrate financial need for medical or travel expenses. Certain programs require patients to be insured and maintain an income level below 400% of the Federal Poverty Level.
Novartis Patient Assistance Foundation
Novartis Patient Assistance Foundation
This fund provides financial assistance to U.S. residents seeking a second opinion from a sarcoma specialist. Grants are awarded four times per year with a maximum amount of $1,000 per consultation.
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Start the conversation →Latest news about Spinal Muscular Atrophy
1 articlesSource: PubMed + NIH RePORTER + openFDA + clinical-journal RSS · last 30 days · disease-tagged at ingest by AI extraction with human QC
See all news about Spinal Muscular Atrophy →Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Spinal Muscular Atrophy
What is Spinal Muscular Atrophy?
Spinal Muscular Atrophy is treated with 3 medications in our database, including Zolgensma, Spinraza, EVRYSDI. 3 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Novartis, Biogen, Genentech.
How is Spinal Muscular Atrophy inherited?
Spinal Muscular Atrophy follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Spinal Muscular Atrophy?
Yes — 20 recruiting clinical trials are currently listed for Spinal Muscular Atrophy on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Spinal Muscular Atrophy?
25 specialists and care centers treating Spinal Muscular Atrophy are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Spinal Muscular Atrophy?
4 FDA-approved treatments and 10 patient support programs are currently tracked on UniteRare for Spinal Muscular Atrophy. See the treatments and support programs sections for copay assistance, eligibility, and contact details.
Frequently asked questions about Spinal Muscular Atrophy
Auto-generated from canonical disease facts (Orphanet, OMIM, ClinicalTrials.gov, openFDA, NPPES). Not a substitute for clinical guidance.
What is Spinal Muscular Atrophy?
Spinal Muscular Atrophy is a rare disease catalogued in international rare-disease ontologies. It is typically inherited as autosomal recessive. Age of onset is generally variable. For verified primary sources, see the UniteRare Spinal Muscular Atrophy page.
How is Spinal Muscular Atrophy inherited?
Spinal Muscular Atrophy follows autosomal recessive inheritance. Genetic counseling is recommended for affected families to understand recurrence risk in offspring and the likelihood of unaffected siblings being carriers. Variants in the underlying gene(s) may be identified via clinical genetic testing.
Are there FDA-approved treatments for Spinal Muscular Atrophy?
Yes — UniteRare tracks 3 FDA-approved treatments with indications relevant to Spinal Muscular Atrophy. Each entry includes prescribing information, orphan-drug-designation status where applicable, and the FDA application number for verification.
Are there clinical trials recruiting for Spinal Muscular Atrophy?
UniteRare currently lists 20 clinical trials relevant to Spinal Muscular Atrophy sourced from ClinicalTrials.gov. Each trial entry includes recruitment status, eligibility criteria summary, principal-investigator information, and study locations. Patients should discuss eligibility with their healthcare provider before enrolling.
How do I find a specialist for Spinal Muscular Atrophy?
UniteRare lists 25 verified clinicians with documented expertise in Spinal Muscular Atrophy, sourced from ClinicalTrials.gov principal-investigator records, PubMed publication histories, and the NPPES NPI registry. Filter by state or browse our state-specific specialist pages for nearby options.
See full Spinal Muscular Atrophy page for complete clinical details, sources, and verified-specialist listings.
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