Double blind trial of a deuterated form of linoleic acid (RT001) in Friedreich ataxia.

Objectives: Friedreich ataxia is (FRDA) an autosomal recessive neurodegenerative disorder associated with intrinsic oxidative damage, suggesting that decreasing lipid peroxidation (LPO) might ameliorate disease progression. The present study tested the ability of RT001, a deuterated form of linoleic acid (D2-LA), to alter disease severity in patients with FRDA in a double-blind placebo-controlled trial.

Methods: Sixty-five subjects were recruited across six sites and received either placebo or active drug for an 11-month study.

Evaluation of Cerebellar Ataxic Patients.

Cerebellar ataxia results from damage to the cerebellum and presents as movement incoordination and variability, gait impairment, and slurred speech. Patients with cerebellar ataxia can also have cognitive and mood changes. Although the identification of causes for cerebellar ataxia can be complex, age of presentation, chronicity, family history, and associated movement disorders may provide diagnostic clues.

Therapies, Research Funding, and Racial Diversity in Essential Tremor: A Systematic Review of the Literature.

Background: Essential tremor (ET) is one of the most common tremor disorders in the world. Despite this, only one medication, propranolol, is approved by the Food and Drug Administration to treat it.

Objectives: We analyzed controlled clinical trials in ET, spanning the last 50 years, to identify potential shortcomings in the therapeutic clinical pipeline.

Current and emerging treatment modalities for spinocerebellar ataxias.

Introduction: Spinocerebellar ataxias (SCA) are a group of rare neurodegenerative diseases that dramatically affect the lives of affected individuals and their families. Despite having a clear understanding of SCA's etiology, there are no current symptomatic or neuroprotective treatments approved by the FDA.

Areas Covered: Research efforts have greatly expanded the possibilities for potential treatments, including both pharmacological and non-pharmacological interventions.

Emerging therapies in Friedreich's Ataxia.

Introduction: Friedreich's ataxia (FRDA) is a progressive, neurodegenerative disease that results in gait and limb ataxia, diabetes, cardiac hypertrophy, and scoliosis. At the cellular level, FRDA results in the deficiency of frataxin, a mitochondrial protein that plays a vital role in iron homeostasis and amelioration of oxidative stress. No cure currently exists for FRDA, but exciting therapeutic developments which target different parts of the pathological cascade are on the horizon.

Orphan Drugs In Development For The Treatment Of Friedreich's Ataxia: Focus On Omaveloxolone.

Friedreich's Ataxia (FRDA) is a devastating and progressive ataxia, marked by mitochondrial dysfunction and oxidative stress. Nrf2 activators such as omaveloxolone (Omav) modulate antioxidative mechanisms, and thus may be viable therapeutic agents in FRDA.

Treatment of cerebellar ataxia.

Symptoms of cerebellar degeneration include ataxia or wide-based gait, visual and speech dysfunction, dysmetria, and dyscoordination. The etiology of cerebellar degeneration is vast and often complex, and requires neuroimaging, lab assessments, and a thorough family history to delineate its cause. There is currently no accepted treatment of hereditary cerebellar degeneration, although several pharmaceutical agents have shown potential promise.

Update on treatment of essential tremor.

Essential tremor is one of the most common movement disorders in the world. Although millions of people worldwide are affected by ET, only one medication, propranolol, is approved by the United States Food and Drug Administration to treat it. None of the medications currently used as ET therapy were developed specifically for this purpose, and select antihypertensive and antiepileptic medications remain at the forefront of ET therapy.