Amicus Therapeutics
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2002-01-01
- Employees
- 517
- Market Cap
- $3.3B
- Website
- http://www.amicusrx.com
- Introduction
Amicus Therapeutics, Inc. operates as a biotechnology company. The firm focuses on discovering, developing and delivering medicines for people living with metabolic diseases. Its product portfolio includes first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants, a novel, clinical-stage, treatment paradigm for Pompe disease and a rare disease gene therapy portfolio. The company was founded by John Francis Crowley on February 4, 2002 and is headquartered in Princeton, NJ.
A Global Prospective Observational Registry of Patients With Pompe Disease
- Conditions
- Interventions
- First Posted Date
- 2023-11-07
- Last Posted Date
- 2024-10-30
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 500
- Registration Number
- NCT06121011
- Locations
- 🇺🇸
Cincinnati Children's Hospital, Cincinnati, Ohio, United States
🇩🇪Universitätsklinikum Gießen und Marburg GmhH, Marburg, Germany
🇺🇸University of Arkansas Medical Science, Little Rock, Arkansas, United States
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
- Conditions
- Interventions
- First Posted Date
- 2021-03-22
- Last Posted Date
- 2024-11-06
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 36
- Registration Number
- NCT04808505
- Locations
- 🇺🇸
University of Florida Clinical Research Center, Gainesville, Florida, United States
🇺🇸The Emory Clinic, Atlanta, Georgia, United States
🇺🇸Duke University Early Phase Research Unit, Durham, North Carolina, United States
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
- First Posted Date
- 2021-03-18
- Last Posted Date
- 2024-04-03
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 44
- Registration Number
- NCT04804566
- Locations
- 🇺🇸
Engage Health, Eagan, Minnesota, United States
Natural History Study of Batten Disease
- First Posted Date
- 2020-11-25
- Last Posted Date
- 2022-04-11
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 10
- Registration Number
- NCT04644549
- Locations
- 🇺🇸
University of Rochester Medical Center, Rochester, New York, United States
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
- Conditions
- First Posted Date
- 2020-03-31
- Last Posted Date
- 2024-08-16
- Lead Sponsor
- Amicus Therapeutics
- Registration Number
- NCT04327973
- Locations
- 🇺🇸
UCSF Benioff Children's Hospital Oakland, Oakland, California, United States
🇺🇸UF Health Shands Hospital, Gainesville, Florida, United States
🇮🇹University Hospital of Padova, Padova, Italy
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
- First Posted Date
- 2020-02-24
- Last Posted Date
- 2024-11-04
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 82
- Registration Number
- NCT04281537
- Locations
- 🇧🇷
Instituto de Genética e Erros Inatos do Metabolismo (IGEIM), São Paulo, Brazil
🇺🇸Amicus Therapeutics, Inc., Philadelphia, Pennsylvania, United States
🇨🇳National Taiwan University, Taipei, Taiwan
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
- First Posted Date
- 2020-02-18
- Last Posted Date
- 2023-02-06
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 10
- Registration Number
- NCT04273243
- Locations
- 🇺🇸
Nationwide Children's Hosptial, Columbus, Ohio, United States
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
- First Posted Date
- 2020-02-05
- Last Posted Date
- 2024-02-08
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 20
- Registration Number
- NCT04252066
- Locations
- 🇺🇸
Amicus Therapeutics, Inc. Pregnancy Registry, Philadelphia, Pennsylvania, United States
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
- Conditions
- Interventions
- First Posted Date
- 2019-10-24
- Last Posted Date
- 2024-11-15
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 110
- Registration Number
- NCT04138277
- Locations
- 🇺🇸
University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
🇺🇸University of California, Irvine, Irvine, California, United States
🇺🇸University of Florida Clinical Research Center, Gainesville, Florida, United States
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) with Fabry Disease
- First Posted Date
- 2019-08-08
- Last Posted Date
- 2024-12-19
- Lead Sponsor
- Amicus Therapeutics
- Target Recruit Count
- 15
- Registration Number
- NCT04049760
- Locations
- 🇺🇸
The Emory Clinic, Atlanta, Georgia, United States
🇺🇸University of Minnesota Masonic Children's Hospital and Clinics, Minneapolis, Minnesota, United States
🇺🇸Lysosomal & Rare Disorders Research & Treatment Center, Fairfax, Virginia, United States