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Displaying 1 - 4 of 4

Molecular Basis of Cardiopulmonary Disorders

Condition: Lung Disease
Investigator: Christine Kim Garcia, MD
Status: Currently Recruiting
The purpose of this study is to identify genetic causes of lung diseases and to develop a research repository so that we can better understand, prevent and treat these conditions. You should consider joining this study if you have been diagnosed with a lung disease that has a genetic cause or if other family members have been diagnosed with a similar lung…
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Study of HMGA2 in Young Women with Lymphangioleiomyomatosis (LAM)

Condition: Lung Disease
Investigator: Jeanine Marie D'Armiento, MD
Status: Currently Recruiting
Lymphangioleiomyomatosis (LAM) is a rare lung disease primarily affecting young women. Currently, LAM is often diagnosed in patients with radiology imaging (CTs) and/or lung biopsies; however, these methods can be imprecise, invasive, and/or can carry significant risks. The current study aims to devise a less invasive tool for diagnosis of this rare…
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Study of ION-682884 (SC) in patients with TTR Amyloidosis Polyneuropathy

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…
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HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

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…
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