224 Studies Now Enrolling
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Displaying 1 - 5 of 5
Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Darryl De Vivo, MD
Status: Closed
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Claudia Chiriboga, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
Condition: Blood Disorders / Amyloidosis
Investigator: Thomas Brannagan, MD
Status: Closed
The purpose of this study is to evaluate the efficacy and safety of ION-682884 given for 24 months in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) also known as Familial Amyloid Polyneuropathy (FAP), and determine if it can help people with mild or moderate polyneuropathy. To be in this study you must have FAP and be…
Condition: Blood Disorders / Amyloidosis
Investigator: Thomas Brannagan, MD
Status: Closed
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…
Condition: Obstetrics & Gynecology / Prenatal Abnormalities
Investigator: Ronald Wapner, MD
Status: Closed
The purpose of this study is to collect blood samples from women who are pregnant with a fetus that has been confirmed by prenatal microarray testing to have a chromosomal abnormality, such as a microdeletion (missing chromosomes) or microduplication (extra chromosomes). The sponsor of this study, Sequenom Laboratories, will use these samples to develop and…