Overview
Also known as:
FDA & Trial Timeline
7 eventsSohag University — NA
Wladimir MAUHIN, Dr
Wladimir MAUHIN, Dr — NA
Chung-Hsing Wang
Sanofi
Albert Einstein College of Medicine — NA
Institut National de la Santé Et de la Recherche Médicale, France
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableXENPOZYME
Treatment of adult patients with non-neurological manifestations of acid sphingomyelinase deficiency (ASMD type B and type A/B). First approved enzyme replacement therapy for ASMD.
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersMount Sinai Hospital
📍 New York, New York
Sohag University Hospital
📍 Sohag
Stanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
Financial Resources
1 resourcesTravel Grants
No travel grants are currently matched to Acid sphingomyelinase deficiency.
Community
No community posts yet. Be the first to share your experience with Acid sphingomyelinase deficiency.
Start the conversation →Latest news about Acid sphingomyelinase deficiency
Disease timeline:
New recruiting trial: National Cohort on Congenital Defects of the Eye
A new clinical trial is recruiting patients for Acid sphingomyelinase deficiency
New recruiting trial: GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
A new clinical trial is recruiting patients for Acid sphingomyelinase deficiency
New recruiting trial: A 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)
A new clinical trial is recruiting patients for Acid sphingomyelinase deficiency
New trial: Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)
Phase NA trial recruiting.
New trial: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Phase NA trial recruiting. Confirmatory Testing
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 Acid sphingomyelinase deficiency
Are there clinical trials for Acid sphingomyelinase deficiency?
Yes — 3 recruiting clinical trials are currently listed for Acid sphingomyelinase deficiency on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Acid sphingomyelinase deficiency?
3 specialists and care centers treating Acid sphingomyelinase deficiency are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Acid sphingomyelinase deficiency?
1 patient support program are currently tracked on UniteRare for Acid sphingomyelinase deficiency. See the treatments and support programs sections for copay assistance, eligibility, and contact details.