Posts by: Alice Melão

News

Reata Pharmaceuticals sold some of its shares, generating sales of approximately $115.9 million to be used to support the clinical development of the company’s pipeline, including omaveloxolone, its lead ... Read more

July 27, 2017

Researchers Identify 2 Molecules That Boost Frataxin Levels in Friedreich’s Ataxia Animal Models

News

Two small molecules were shown to increase the mRNA and protein levels of frataxin (FXN) in animal models of Friedreich’s ataxia. According to a report published at Neuropharmacology, these compounds could have ... Read more

June 13, 2017

Multiple Sclerosis Treatment Holds Promise for Friedreich’s Ataxia Patients, Studies Show

Friedreich's Ataxia, News

Friedreich’s ataxia (FRDA) patients have less mitochondria than other people because of the mutation in the frataxin (FXN) gene that is associated with the disorder, according to two studies. ... Read more

April 11, 2017

Discovery Could Address Reduced Protein Expression Associated with Friedreich’s Ataxia

News

Friedreich’s ataxia (FRDA) stems from abnormal DNA code expansions in the Frataxin (FXN) gene that collectively reduce the expression of the FXN protein. A new study indicates that small RNA ... Read more

April 6, 2017April 7, 2017

Voyager Therapeutics’ Potential Gene Therapy Program for FA Moving Toward Pre-Clinical Stage

News

Voager Therapeutics has announced that its Friedreich’s ataxia program, VY-FXN01, is taking steps to initiate preclinical tests of its safety and effectiveness, the company expects to achieve in a ... Read more

March 30, 2017March 30, 2017

Identifying Cerebellum’s Structural Changes May Serve as Biomarker for Ataxia, Study Finds

News

Researchers at Italy’s IRCCS Santa Lucia Foundation have found that cerebellar atrophy can affect brain structures related to emotions, thinking and memory, which may in part explain ataxia symptoms. Their results suggest that ... Read more

February 7, 2017

Inflammatory Bowel Disease, Growth Hormone Deficiency More Likely in Patients With FA, Study Shows

News

Patients with Friedreich ataxia (FA) are more likely to develop inflammatory bowel disease and growth hormone deficiency than those without FA, concludes a recent study. Researchers believe that these associations, which may ... Read more

December 8, 2016

Altered Cellular Metabolism in Friedreich’s Ataxia Is Potential Therapeutic Target, Review Says

News

Recent research in Friedreich’s ataxia (FA) has revealed new possibilities for lipid metabolism as a therapeutic target to slow the progression of the disease in patients. The latest knowledge ... Read more

November 10, 2016

HFE Gene Mutation May Be Culprit in Peripheral Nerve Damage to Friedrich’s Ataxia Patients

News

An Indian population study has shown that a specific mutation in a gene involved in iron regulation, the HFE gene, is seen frequently in patients with Friedreich’s ataxia (FA), ... Read more

October 27, 2016October 27, 2016

Recognizing Abnormal Movements in Kids Important for Early Diagnosis, Treatment of Ataxia Syndromes

News

Recognizing atypical involuntary movements in childhood autosomal recessive ataxia syndromes can lead to early diagnosis and may allow for more effective treatment of the symptoms, according to a recent ... Read more