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Ultragenyx Pharmaceutical (RARE)
NASDAQ:RARE
No Intervention, Gtx-102
Angelman Syndrome
Phase II
Recruiting
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
Aug 21, 2025
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Setrusumab
Osteogenesis Imperfecta
Phase III
Active Not Recruiting
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
Oct 08, 2024
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Gtx-102, Sham-Lp
Angelman Syndrome
Phase III
Active Not Recruiting
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Sep 24, 2024
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Gtx-102
Angelman Syndrome
Phase III
Enrolling By Invitation
Long-term Extension of GTX-102 in Angelman Syndrome
May 10, 2024
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Triheptanoin, Mct Oil
Long-Chain Fatty Acid Oxidation Disorders (Lc-Faod)
Phase III
Active Not Recruiting
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Jun 27, 2023
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Bisphosphonate, Setrusumab
Osteogenesis Imperfecta
Phase III
Active Not Recruiting
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
Mar 03, 2023
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Oral Corticosteroids, Dtx301, Sodium Acetate
Otc Deficiency
Phase III
Active Not Recruiting
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
Apr 18, 2022
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Oral Corticosteroids, Dtx401
Glycogen Storage Disease Type Ia
Phase III
Active Not Recruiting
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Jul 14, 2021
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Standard Of Care (Soc), Ux701
Wilson Disease
Phase I/II
Active Not Recruiting
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
May 07, 2021
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Adjuvant Immunomodulatory (Im) Therapy
Mps Iiia, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis Iiia
Phase III
Enrolling By Invitation
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
Apr 20, 2020
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FAQ
What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases:
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does Ultragenyx Pharmaceutical (RARE) have in its pipeline
RARE is currently developing the following drugs: No Intervention, Gtx-102, Setrusumab, Gtx-102, Sham-Lp. These drug candidates are in various stages of clinical development as the company works toward FDA approval.