Children's Hospital of the King's Daughters | Novel Therapeutics and Gene Therapy

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD...

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD....

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants w...

The main purpose of this study is to evaluate the long-term efficacy of mirikizumab in pediatric participants with ulcerative colitis (UC) or Crohn's...

This phase II trial studies the side effects and how well nirogacestat works in treating patients less than 18 years of age with desmoid tumors that...

The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atr...

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion o...

The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy...

This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of...

This is a Phase 1-2, multicenter, international, single-arm, open-label study designed to identify a recommended dose of bosutinib administered orall...

The participants of this study will be children, adolescents, and young adults with residual osteosarcoma, which cannot be removed completely through...

This Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spina...

Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory...

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with on...

The purpose of this study is to test the long-term safety of rimegepant in the acute treatment of migraine in children and adolescents (≥ 6 to \< 18...

This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofi...

A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with st...

This is a Phase 1/2a, open-label, single-dose, PK study of VALTOCO with open-label safety period in pediatric epilepsy subjects age 2 to 5 years.

The overall goal of this study is to determine the clinical benefit and safety of antiviral therapy for asymptomatic congenital cytomegalovirus (cCMV...

The purpose of this study is to test the safety and efficacy of BHV-3000 versus placebo in the acute treatment of moderate or severe migraine in chil...