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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). Patients will be treated sequentially in 2…

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

This phase III trial uses the Decipher risk score to guide therapy selection. Decipher score is based on the activity of 22 genes in prostate tumor and may predict how likely it is for recurrent prostate cancer to spread (metastasize) to other parts…

Investigator

Paul L Crispen

Ages

18 Years - N/A

Sexes

Male

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 dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study

Investigator

Barry J Byrne

Ages

2 Years - 3 Years

Sexes

Male

This is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria…

Investigator

Roberto T Zori

Ages

18 Years - 65 Years

Sexes

All