Clinical trials

Development of a central repository for PD-related genomic data for future research.

Investigator

Irene A Malaty

Ages

18 Years - N/A

Sexes

All

The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5).

Investigator

Barry J Byrne

Ages

6 Years - 13 Years

Sexes

All

The purpose of the study is to learn from the real world practice of prescribing targeted therapies to patients with advanced cancer whose tumor harbors a genomic variant known to be a drug target or to predict sensitivity to a drug. Note: Due to…

Investigator

Thomas J George

Ages

12 Years - N/A

Sexes

All

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the…

Investigator

Carmen Leon Astudillo

Ages

N/A - N/A

Sexes

Male

The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized…

Investigator

Barry J Byrne

Ages

4 Years - 7 Years

Sexes

Male

Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of…

Investigator

Tung T Wynn

Ages

2 Years - N/A

Sexes

Male