天使综合征药物研究进展汇总
clinicaltrials
- A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
- HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
- A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
- Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
- A Study in Adults and Adolescents With Angelman Syndrome (STARS)
- An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
- Italian Angelman Syndrome Registry Protocol
- REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
- Parent and Infant Inter(X)Action Intervention (PIXI)
- Early Check: Expanded Screening in Newborns
- Angelman Syndrome Natural History Study
- A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
- A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.
- An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
- Use of Eye Tracking to Study Social Perception Abnormalities in Children with Angelman Syndrome
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