Casimersen
- Generic Name
- Casimersen
- Brand Names
- Amondys 45
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1422958-19-7
- Unique Ingredient Identifier
- X8UHF7SX0R
- Background
Duchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive impairment of ambulatory, pulmonary, and cardiac function and is invariably fatal. A related, albeit a less severe, form of muscular dystrophy known as Becker muscular dystrophy (BMD) is characterize...
- Indication
Casimersen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients confirmed to have a DMD gene mutation amenable to exon 45 skipping. This indication represents an accelerated approval based on observed efficacy; continued approval for this indication may be contingent on the verification of safety and efficacy in a confirmatory tria...
- 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
- 🇺🇸
Arkansas Children's Hospital Research Institute, Little Rock, Arkansas, United States
🇺🇸Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸University 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
- 🇺🇸
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
- 🇮🇹
Fondazione Policlinico Universitario A Gemelli, Milano, Italy
🇧🇪UZ Leuven, Leuven, Belgium
🇺🇸Children's Hospital of Wisconsin, Corporate Center Suite 540, Milwaukee, Wisconsin, United States
Drug Development Updates
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