224 Studies Now Enrolling
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Displaying 1 - 7 of 7
Research Study of New Treatment Called NT-501 (Encapsulated Cell Therapy (ECT) Implant) for Glaucoma
Condition: Eye / Glaucoma
Investigator: George Cioffi, MD
Status: Closed
Glaucoma is a given name of a group of diseases that affect the retinal ganglion cells in the eye and the optic nerve. Glaucoma cause degeneration of the retinal ganglion cells in the retina and their projections (axons) that form the optic nerve. Retinal ganglion cells and their axons transmit all of our vision information from the eye to the brain through…
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: Eye / Glaucoma
Investigator: Lama Al-Aswad, MD, MPH
Status: Closed
This study is for patients with Glaucoma. The purpose of this study is to investigate the safety and effectiveness of a study drug called Bimatoprost Sustained Release (SR) compared to treatment with eye drug Timolol. The Bimatoprost SR is an implant that is very small, is biodegradable (dissolves naturally in the body), and contains the bimatoprost drug.
Condition: Eye / Glaucoma
Investigator: Jeffrey Liebmann, MD
Status: Closed
Glaucoma is the leading cause of irreversible blindness worldwide. The most important test to detect progression is visual field testing. However, this test is very subjective, often unreliable, and variable. One of the main causes of unreliable tests is the lack of attentiveness or concentration during the test. Previous studies have shown that listening…