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FARAFARA Cure FA

 

Scientific News

FARA funds research progress

In this section, you will find the most recent FA research publications, many of which are funded by FARA, as well as information on upcoming conferences and symposiums. You can search for articles by date using the archive box in the right hand column. To locate FARA Funded or Supported Research, click the hyperlink in the right hand column. You may also search for specific content using key words or phrases in the search button at the top right of your screen. Please be sure to visit other key research sections of our website for information on FARA’s Grant Program and the Treatment Pipeline.

 


 

Edison Pharmaceuticals Announces FDA Grants EPI-A0001

Orphan Drug Designation for Inherited Mitochondrial Respiratory Chain Diseases

Edison Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EPI-A0001 for treatment of inherited mitochondrial respiratory chain diseases. These mitochondrial diseases today affect an estimated
60,000 individuals.

Read more: Edison Pharmaceuticals Announces FDA Grants EPI-A0001

Idebenone Phase II Trial Reaches Midpoint at National Institutes of Health

Washington, D.C. — National Institutes of Health (NIH) researcher Dr. Nicholas Di Prospero has reported that his team has reached the midpoint of its phase II clinical trial of Idebenone at the National Institute of Neurological Disorders and Stroke (NINDS).

Dr. Di Prospero’s team has examined and tested each of the 48 Friedreich’s ataxia patients enrolled in the phase II clinical trial of Idebenone at NINDS which is part of NIH. The same patients will begin returning for their second and final visits (six months after their first). The second visits will conclude in August and the phase II trial will be over. Dr. Di Prospero’s team will then analyze and publish the results of the study.

Dr. Di Prospero and the NINDS team indicated they want to thank all of the families that expressed interest in the trial, those that sought enrollment and those that have participated. On behalf of all Friedreich's ataxia families everywhere, FARA would like to add its own gratitude to Dr. Di Prospero, the fine NINDS team and to the participating families for making this important clinical trial possible and for keeping it on schedule and on track.

Contact

Ronald Bartek
President, Friedreich’s Ataxia Research Alliance
(703) 413-4468
fara@FAResearchAlliance.org

Austrian Lab Supported by FARA Publishes Exciting Findings

Drug Increases Frataxin Protein Levels Two to Five Fold in Cells from Friedreich's Ataxia Patients

Abstract

Background

Friedreich's ataxia (FRDA) is a neurodegenerative disorder caused by decreased expression of the protein frataxin, recently described to be an iron chaperone for the assembly of iron-sulphur clusters in the mitochondria, causing iron accumulation in mitochondria, oxidative stress and cell damage. Searching for compounds that could possibly influence frataxin expression, we found that the cytokine recombinant human erythropoietin (rhuEPO) significantly increases frataxin expression by a still unknown mechanism.

Read more: Austrian Lab Supported by FARA Publishes Exciting Findings

Santhera starts European Phase III Study with its Lead Compound SNT-MC17 (idebenone) in Friedreich’s Ataxia

Santhera Pharmaceuticals AG (“Santhera”), a Swiss-based biopharmaceutical company focused on neuromuscular diseases, announced today that it has started a European Phase III clinical study with its lead product SNT-MC17 (idebenone) in Friedreich’s Ataxia (FRDA). The design of this study reflects the company’s discussions with EMEA which took place earlier this year and will evaluate both the cardiacand neurological benefits of SNT-MC17 (idebenone).

Read more: Santhera starts European Phase III Study with its Lead Compound SNT-MC17 (idebenone) in...

Santhera and the NIH Collaborate to Evaluate SNT-MC17 in FA

Santhera Pharmaceuticals AG (“Santhera”) of Switzerland announced today the start of a collaborative clinical trial with the US National Institutes of Neurological Disorders and Stroke (NINDS) at the National Institute of Health (NIH) to evaluate SNT-MC17 (idebenone) in patients affected by Friedreich’s ataxia (FRDA), a devastating life-threatening neuromuscular disease.

Read more: Santhera and the NIH Collaborate to Evaluate SNT-MC17 in FA
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