Clinical trials

Investigator

Manuel A Amaris

BLOCK-SAH is a phase II, multicenter, randomized, double-blinded, placebo-controlled clinical trial with a sequential parallel comparison design (SPCD) of bilateral pterygopalatine fossa (PPF) injections with 20mg ropivacaine + 4mg dexamethasone…

Investigator

Carolina B Maciel

Ages

18 Years - 85 Years

Sexes

All

Investigator

Carolina B Maciel

The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).

Investigator

Mustafa M Ahmed

Ages

8 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

This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study…

Investigator

Sub Subramony

Ages

10 Years - 55 Years

Sexes

All