Overview
Mucopolysaccharidosis is treated with 1 medication in our database, including MEPSEVII. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Ultragenyx. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Mucopolysaccharidosis treatment below.
FDA & Trial Timeline
10 eventsAssiut University — NA
Denali Therapeutics Inc. — PHASE2, PHASE3
Immusoft of CA, Inc. — PHASE1
JCR Pharmaceuticals Co., Ltd. — PHASE3
GC Biopharma Corp — PHASE1
Denali Therapeutics Inc. — PHASE2, PHASE3
University of California, San Francisco — NA
JCR Pharmaceuticals Co., Ltd. — PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
1 availableMEPSEVII
* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: Not Publicly Available * Core Eligibility Restrictions: Not Publicly Available * RxBIN, PCN, and Group numbers: Not Publi
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersChildren's Hospital of Philadelphia
📍 Philadelphia, Pennsylvania
👤 Study Director
👤 Richard Neibeger, MD
UCSF Benioff Children's Hospital Oakland
📍 Oakland, California
👤 Neeta Thakur, MD, MPH
University of California San Francisco
📍 San Francisco, California
Hôpital Femme Mère Enfant
📍 Bron
👤 Justine BACCHETTA, MD PhD
Baylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
3 grantsThe Assistance Fund (TAF) Financial Assistance Program
Applicants must be U.S. citizens or permanent residents diagnosed with MPS II (Hunter Syndrome) and prescribed an FDA-approved treatment with existing insurance coverage. Financial eligibility is required based on household income and size, though specific thresholds are not listed.
Community
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Start the conversation →Latest news about Mucopolysaccharidosis
Disease timeline:
New recruiting trial: A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
A new clinical trial is recruiting patients for Mucopolysaccharidosis
New recruiting trial: Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
A new clinical trial is recruiting patients for Mucopolysaccharidosis
New recruiting trial: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III
A new clinical trial is recruiting patients for Mucopolysaccharidosis
New recruiting trial: Study of Skeletal Disorders
A new clinical trial is recruiting patients for Mucopolysaccharidosis
New recruiting trial: Decoding the Genetic Landscape of Skeletal Diseases
A new clinical trial is recruiting patients for Mucopolysaccharidosis
New trial: Registry of Patients Diagnosed With Lysosomal Storage Diseases
Phase NA trial recruiting. There is no intervention
New trial: An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrom
Phase PHASE3 trial recruiting. JR-141
New trial: An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Phase PHASE2 trial recruiting. JR-141
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 Mucopolysaccharidosis
What is Mucopolysaccharidosis?
Mucopolysaccharidosis is treated with 1 medication in our database, including MEPSEVII. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Ultragenyx. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Mucopolysaccharidosis treatment below.
Are there clinical trials for Mucopolysaccharidosis?
Yes — 16 recruiting clinical trials are currently listed for Mucopolysaccharidosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Mucopolysaccharidosis?
21 specialists and care centers treating Mucopolysaccharidosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Mucopolysaccharidosis?
1 FDA-approved treatment and 1 patient support program are currently tracked on UniteRare for Mucopolysaccharidosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.