Vosoritide
- Generic Name
- Vosoritide
- Brand Names
- Voxzogo
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1480724-61-5
- Unique Ingredient Identifier
- 7SE5582Q2P
- Background
Achondroplasia is an autosomal dominant genetic disease and the most common cause of dwarfism in humans. It results from a gain-of-function missense mutation in FGFR3 that results in a dramatic suppression of bone growth, both in volume and in length. Treatment for achondroplasia includes both surgical and pharmacological interventions, the latter of which i...
- Indication
Vosoritide is indicated for the promotion of linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses.
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- Associated Conditions
- -
- Associated Therapies
- -
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
- First Posted Date
- 2024-06-12
- Last Posted Date
- 2024-06-12
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 80
- Registration Number
- NCT06455059
- Locations
- 🇺🇸
Children's National Medical Center, Washington, District of Columbia, United States
🇦🇺Murdoch Children's Research Institute, Parkville, Victoria, Australia
Vosoritide for Short Stature in Turner Syndrome
- First Posted Date
- 2023-05-08
- Last Posted Date
- 2024-06-24
- Lead Sponsor
- Roopa Kanakatti Shankar, MBBS, MS
- Target Recruit Count
- 20
- Registration Number
- NCT05849389
- Locations
- 🇺🇸
Children's National Hospital, Washington, District of Columbia, United States
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
- First Posted Date
- 2020-09-18
- Last Posted Date
- 2024-10-09
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 20
- Registration Number
- NCT04554940
- Locations
- 🇦🇺
Murdoch Children's Research Institute, Parkville, Victoria, Australia
🇬🇧Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital, London, United Kingdom
🇬🇧Sheffield Children's NHS Foundation Trust, Sheffield, United Kingdom
Vosoritide for Selected Genetic Causes of Short Stature
- First Posted Date
- 2020-01-06
- Last Posted Date
- 2024-02-06
- Lead Sponsor
- Andrew Dauber
- Target Recruit Count
- 56
- Registration Number
- NCT04219007
- Locations
- 🇺🇸
Children's National Hospital, Washington, District of Columbia, United States
Drug Development Updates
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