Spinocerebellar Ataxia Overview
Learn About Spinocerebellar Ataxia
View Main Condition: Movement Disorders
State University Of Iowa
Katherine Mathews is a Neurologist and a Pediatrics provider in Iowa City, Iowa. Dr. Mathews is rated as an Elite provider by MediFind in the treatment of Spinocerebellar Ataxia. Her top areas of expertise are Limb-Girdle Muscular Dystrophy Type 2I, Limb-Girdle Muscular Dystrophy, Dystrophinopathy, and Friedreich Ataxia. Dr. Mathews is currently accepting new patients.
The Regents Of The University Of California
Susan Perlman is a Neurologist in Los Angeles, California. Dr. Perlman is rated as an Elite provider by MediFind in the treatment of Spinocerebellar Ataxia. Her top areas of expertise are Spinocerebellar Ataxia, Friedreich Ataxia, Drug Induced Dyskinesia, and Olivopontocerebellar Atrophy.
Massachusetts General Physicians Organization Inc
Jeremy Schmahmann is a Neurologist in Boston, Massachusetts. Dr. Schmahmann is rated as an Elite provider by MediFind in the treatment of Spinocerebellar Ataxia. His top areas of expertise are Spinocerebellar Ataxia, Acute Cerebellar Ataxia, Olivopontocerebellar Atrophy, Drug Induced Dyskinesia, and Gastrostomy. Dr. Schmahmann is currently accepting new patients.
Summary: Spinocerebellar ataxias 27B (SCA27B) is caused by an expansion of ≥ 250 GAA triplets in the FGF14 gene and accounts for 15% of cerebellar ataxias (around 500 patients in France). It is a late-onset form often presenting paroxysmal episodes of ataxia and/or diplopia. The disease progresses slowly, with an average increase of 0.10 points/year on the Friedreich's Ataxia Rating Scale (FARS) - Function...
Summary: This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.