Thursday, April 26, 2018

BMN 290 for Friedreich's Ataxia

›
SAN RAFAEL, Calif., April 25, 2018 /PRNewswire In the fourth quarter of 2017, BioMarin announced that it had selected as its next clinical...
Wednesday, April 25, 2018

Reata Announces New Preclinical Data Demonstrating the Potential of Omaveloxolone in the Treatment of Friedreich’s Ataxia and Other Severe Neurological Diseases

›
IRVING, Texas, April 24, 2018 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq:RETA) (Reata or Company), a clinical-stage biopharmace...

Patient reported outcome measures in rare diseases: a narrative review

›
Anita Slade, Fatima Isa, Derek Kyte, Tanya Pankhurst, Larissa Kerecuk, James Ferguson, Graham Lipkin and Melanie Calvert, Orphanet Journal o...

Emergence of breath testing as a new non-invasive diagnostic modality for neurodegenerative diseases

›
N. Siva Subramaniam, C.S. Bawden, H. Waldvogel, R.M.L. Faull, G.S. Howarth, R.G. Snell, , Brain Research, Available online 22 April 2018, IS...
Tuesday, April 24, 2018

The idebenone metabolite QS10 restores electron transfer in complex I and coenzyme Q defects

›
Valentina Giorgio, Marco Schiavone, Chiara Galber, Marco Carini, Tatiana Da Ros, Valeria Petronilli, Francesco Argenton, Valerio Carelli, Ma...
Sunday, April 22, 2018

GoFAR reinforces partnership with Powell Gene Therapy Center at University of Florida (UF) to develop a gene therapy for Friedreich’s ataxia

›
Torino-Italia, April 20-2018. In January, GoFAR awarded an additional grant of $450,000 to Manuela Corti, P.T., Ph.D., assistant professor o...
Friday, April 20, 2018

Retrotope Announces Peer-Reviewed Publication of Positive Phase 1b/2a Findings for RT001 in Friedreich’s Ataxia

›
Retrotope, April 18, 2018. Protocol for Pivotal Clinical Trial Has Been Submitted to the US FDA LOS ALTOS, Calif., April 18, 2018 (GLOBE N...
Wednesday, April 18, 2018

Could stem cell therapy be an effective treatment for Friedreich’s ataxia?

›
University of Bristol, Press release issued: 17 April 2018 Researchers at the University of Bristol are looking for people with an inherit...
Tuesday, April 17, 2018

Could statins ease deadly heart condition in rare neuromuscular disease?

›
AAS and EurekAlert, Public Release: 17-Apr-2018.  University of Pennsylvania School of Medicine. PHILADELPHIA - In preclinical studies usin...
Wednesday, April 11, 2018

Mitochondria in the nervous system: From Health to disease, part II

›
Maria Teresa Carrì, Brian M. Polster, Philip M. Beart, Mitochondria in the nervous system: From Health to disease, part II, Neurochemistry I...