Acid sphingomyelinase deficiency Advocacy Hub | UniteRare

At a Glance

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Regulatory Watchboard

7 events

Nov 2025Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)

Sohag University — NA

TrialACTIVE NOT RECRUITING

May 2025National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

Wladimir MAUHIN, Dr

TrialNOT YET RECRUITING

May 2025Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease

Wladimir MAUHIN, Dr — NA

TrialNOT YET RECRUITING

May 2024Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Chung-Hsing Wang

TrialNOT YET RECRUITING

Apr 2024A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

Sanofi

TrialRECRUITING

May 2021ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Albert Einstein College of Medicine — NA

TrialENROLLING BY INVITATION

Jul 2017National Cohort on Congenital Defects of the Eye

Institut National de la Santé Et de la Recherche Médicale, France

TrialRECRUITING

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

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

Total programs

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XENPOZYME

Sanofi

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Approved Treatments

1 FDA-approved

XENPOZYME

(OLIPUDASE ALFA-RPCP)Orphan drug

Genzyme Corporation

FDA label ↗

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Clinical Trial Landscape

3 active trials

2N/A

1Unknown

3Total recruiting

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Recent News & Research

No recent news articles indexed yet for Acid sphingomyelinase deficiency.
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Specialist Network

Top 3 by expertise

Melissa Wasserstein, MD

Maimonides Medical Center

📍 Brooklyn, New York

Score 13

Wladimir MAUHIN, Doctor

Groupe Hospitalier Diaconesses Croix Saint-Simon

📍 Paris, France

Score 32 trials

Carla EM Hollak

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Score 02 trials

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