Distal hereditary motor neuropathy Advocacy Hub | UniteRare

At a Glance

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Regulatory Watchboard

No FDA or trial events recorded yet for Distal hereditary motor neuropathy.

Financial Assistance

4 programs

FINANCIAL LANDSCAPE SUMMARY

Total programs

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Patient Assistance Programs4

Spinraza

Biogen Inc.

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Zolgensma

Novartis Gene Therapies, Inc.

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ITVISMA

Novartis Gene Therapies, Inc.

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EVRYSDI

Genentech, Inc.

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Approved Treatments

4 FDA-approved

ITVISMA

(ONASEMNOGENE ABEPARVOVEC-BRVE)Orphan drug

Novartis Gene Therapies, Inc.

Approved Nov 2025FDA label ↗

EVRYSDI

(RISDIPLAM)Orphan drug

Genentech, Inc.

Approved Aug 2020FDA label ↗

Zolgensma

(ONASEMNOGENE ABEPARVOVEC-XIOI)Orphan drug

Novartis Gene Therapies, Inc.

Approved May 2019FDA label ↗

Spinraza

(NUSINERSEN)Orphan drug

Biogen Inc.

Approved Dec 2016FDA label ↗

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Clinical Trial Landscape

No active clinical trials currently recruiting for Distal hereditary motor neuropathy.
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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.

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Specialist Network

Top 6 by expertise

Chiara Marini-Bettolo, MD, PhD

Newcastle University

📍 Newcastle upon Tyne

Score 83 trials

Matthew ER Butchbach, Ph.D.

Nemours Children's Clinic

Score 22 trials

Philippe Latour

Service de Biochimie et Biologie Moléculaire

Score 03 pubs

Byung-Ok Choi

Samsung Advanced Institute for Health Sciences & Technology

Score 03 pubs

Ki Wha Chung

Kongju National University

Score 03 pubs

Tanya Stojkovic

Nord/Est/Ile-de-France Neuromuscular Diseases Reference Centre

Score 04 pubs

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