Golodirsen
- Generic Name
- Golodirsen
- Brand Names
- Vyondys 53
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1422959-91-8
- Unique Ingredient Identifier
- 033072U4MZ
- Background
Golodirsen is a morpholino antisense oligomer designed to treat about 8% of patients with Duchenne Muscular Dystrophy (DMD). This is an X-linked condition leading to progressive muscle degeneration that begins in early childhood, rendering many patients wheelchair-bound by age 12. Often, patients succumb to this condition by age 30 or younger due to cardiac ...
- Indication
Golodirsen is indicated to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that would benefit from exon 53 skipping. Continued FDA approval of this drug is contingent upon the results of clinical trials to confirm its benefit.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
- Conditions
- Interventions
- First Posted Date
- 2024-09-23
- Last Posted Date
- 2024-09-23
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 300
- Registration Number
- NCT06606340
- Locations
- ๐บ๐ธ
Arkansas Children's Hospital Research Institute, Little Rock, Arkansas, United States
๐บ๐ธChildren's Hospital Los Angeles, Los Angeles, California, United States
๐บ๐ธUniversity of California Davis Medical Center, Sacramento, California, United States
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
- Conditions
- Interventions
- First Posted Date
- 2019-11-27
- Last Posted Date
- 2023-10-26
- Lead Sponsor
- Kevin Flanigan
- Target Recruit Count
- 3
- Registration Number
- NCT04179409
- Locations
- ๐บ๐ธ
Nationwide Children's Hospital, Columbus, Ohio, United States
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
- First Posted Date
- 2018-05-22
- Last Posted Date
- 2023-08-21
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Target Recruit Count
- 171
- Registration Number
- NCT03532542
- Locations
- ๐ฎ๐น
Fondazione Policlinico Universitario A Gemelli, Milano, Italy
๐ง๐ชUZ Leuven, Leuven, Belgium
๐บ๐ธChildren's Hospital of Wisconsin, Corporate Center Suite 540, Milwaukee, Wisconsin, United States
Drug Development Updates
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