Studies led by investigators in the Division of Autonomic Disorders aim to advance our ability to effectively treat genetic disorders, synucleinopathies, and multiple system atrophy, among other conditions.

Genetic Disorders

Natural History of Familial Dysautonomia (FD)
Principal investigator: Horacio Kaufmann, MD

Natural History of Congenital Insensitivity to Pain with Anhidrosis (CIPA)
Principal investigator: Horacio Kaufmann, MD

Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Generation of Disease Models with Induced Pluripotent Stem Cells (iPSCs) in Hereditary Sensory and Autonomic Neuropathies
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Synucleinopathies

Natural History Study of Synucleinopathies
Principal investigator: Horacio Kaufmann, MD

Multiple System Atrophy

Clinical Trial Readiness for Multiple System Atrophy (MSA)
Principal investigator: Horacio Kaufmann, MD

The Global Multiple System Atrophy Registry (GLoMSAR)
Principal Investigator: Horacio Kaufmann, MD

TRACK-MSA
Principal investigator: Horacio Kaufmann, MD

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic nOH in Participants With Multiple System Atrophy (CYPRESS)
Principal Investigator: Patricio A. Millar Vernetti, MD

AMULET—Delay Multiple System Atrophy
Principal investigator: Patricio A. Millar Vernetti, MD

UB-312 in Patients With Synucleinopathies
Principal Investigator: Horacio Kaufmann

A Study of TAK-341 in Treatment of Multiple System Atrophy
Principal Investigator: Patricio A. Millar Vernetti, MD

A Phase 2, double-blind, placebo-controlled, parallel-group study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential efficacy of multiple doses of ONO-2808 in patients with Multiple System Atrophy (MSA)
Principal investigator: Patricio A. Millar Vernetti, MD

ATTR Amyloidosis

Autonomic dysfunction and peripheral neuropathy in patients with cardiac ATTR amyloidosis
Principal investigator: Alejandra Gonzalez-Duarte Briseno, MD

Contact Us

To learn more about our clinical trials or to participate, please call Jose Martinez, senior clinical trials manager, at 212-263-7225.