Spinal Muscular Atrophy Advocacy Hub | UniteRare

At a Glance

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⚡ Live Alerts

PAN Foundation — Spinal Muscular Atrophy status changedApply now ↗

Regulatory Watchboard

10 events

Apr 2026Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular Atrophy

Charles University, Czech Republic — NA

TrialNOT YET RECRUITING

Mar 2026SPINRAZA: New indication approved

FDAcompleted

Mar 2026HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness

Teachers College, Columbia University — NA

TrialRECRUITING

Mar 2026Gait and Bone Health in SMA

Jacqueline Montes

TrialRECRUITING

Feb 2026EVRYSDI: New indication approved

FDAcompleted

Feb 2026EVRYSDI: New indication approved

FDAcompleted

Jan 2026EVRYSDI: New indication approved

FDAcompleted

Jan 2026EVRYSDI: New indication approved

FDAcompleted

Jan 2026Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Gemma Biotherapeutics — PHASE1, PHASE2

TrialRECRUITING

Jan 2026Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers

Institut de Myologie, France — NA

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

24 programs

FINANCIAL LANDSCAPE SUMMARY

Total programs

Open now

$6K

Available/year

Foundation grants

Copay cards

Travel grants

Foundation Grants7

Good Days — Spinal Muscular Atrophy

Good Days

OpenContact for detailsApply ↗

PAN Foundation — Spinal Muscular Atrophy

PAN Foundation

ClosedContact for detailsApply ↗

Patient Services Inc — Spinal Muscular Atrophy

Patient Services Inc

OpenContact for detailsApply ↗

NORD Patient Assistance — Spinal Muscular Atrophy

NORD Patient Assistance

OpenContact for detailsApply ↗

Patient Advocate Foundation — Spinal Muscular Atrophy

Patient Advocate Foundation

OpenContact for detailsApply ↗

HealthWell Foundation — Spinal Muscular Atrophy

HealthWell Foundation

OpenContact for detailsApply ↗

The Assistance Fund — Spinal Muscular Atrophy

The Assistance Fund

OpenContact for detailsApply ↗

Copay Assistance2

EVRYSDI

Genentech

OpenContact for detailsApply ↗

Spinraza

Biogen

OpenContact for detailsApply ↗

Patient Assistance Programs1

ITVISMA

Novartis Gene Therapies, Inc.

OpenContact for detailsApply ↗

Travel Grants14

Help Hope Live Financial Assistance for Spinal Muscular Atrophy

Help Hope Live

OpenContact for detailsApply ↗

Help Hope Live Spinal Muscular Atrophy Financial Assistance

OpenContact for detailsApply ↗

Novartis ACCESS Program

Novartis

Contact for detailsApply ↗

Novartis Patient Assistance Foundation

OpenUp to $1K/yrApply ↗

Novartis Patient Assistance Foundation (NPAF)

Novartis Patient Assistance Foundation

OpenUp to $5K/yrApply ↗

Novartis Patient Assistance Foundation, Inc. (NPAF)

Novartis Patient Assistance Foundation, Inc.

Contact for detailsApply ↗

Novartis Patient Assistance Now Oncology (PANO)

Novartis

Contact for detailsApply ↗

Novartis Patient Assistance Program

Novartis

Contact for detailsApply ↗

Novartis Patient Support

Novartis

OpenContact for detailsApply ↗

PAN Foundation Spinal muscular atrophy copay grant

PAN Foundation

ClosedContact for detailsApply ↗

PAN Foundation Spinal Muscular Atrophy Copay Grant

PAN Foundation

Contact for detailsApply ↗

PAN Foundation Spinal Muscular Atrophy Financial Assistance

PAN Foundation

OpenContact for detailsApply ↗

Spinal Muscular Atrophy Copay Grant

PAN Foundation

ClosedContact for detailsApply ↗

Zolgensma Global Managed Access Program (gMAP)

Novartis

ClosedContact for detailsApply ↗

View all support programs on disease page →

Approved Treatments

3 FDA-approved

Zolgensma

(ONASEMNOGENE ABEPARVOVEC-XIOI)Orphan drug

Novartis Gene Therapies, Inc.

FDA label ↗

Spinraza

(NUSINERSEN)Orphan drug

Biogen Inc.

FDA label ↗

EVRYSDI

(RISDIPLAM)Orphan drug

Genentech, Inc.

FDA label ↗

View full treatment details on disease page →

Clinical Trial Landscape

54 active trials

7Phase 3

2Phase 4

3Phase 2

27N/A

9Unknown

5PHASE1, PHASE2

1EARLY_PHASE1

54Total recruiting

Search clinical trials for Spinal Muscular Atrophy →

Recent News & Research

1 article

Lancet NeurologySep 30, 2025

Zolgensma Long-Term Data: 5-Year Outcomes in SMA

Five-year follow-up of Zolgensma in SMA type 1 shows patients maintaining motor milestones with no new safety signals.

Read ↗

Browse all Spinal Muscular Atrophy news →

Specialist Network

Top 6 by expertise

Giorgia Coratti, PhD

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

📍 Roma

Score 31

Maryam Oskoui

Alberta Children's Hospital

📍 Calgary, Alberta

Score 3119 pubs

Eugenio Mercuri, MD

Istituto Gaslini

📍 Genova

Score 313 trials

Sitra Tauscher-Wisniewski, MD

Stanford University Medical Center

📍 Palo Alto, California

Score 21

Pascale Saugier-Veber, PharmD PhD

CHU Rouen

📍 Rouen

Score 19

Chiara Marini-Bettolo, MD, PhD

Newcastle University

📍 Newcastle upon Tyne

Score 83 trials

View all Spinal Muscular Atrophy specialists →

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Financial Assistance

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