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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.


Treatment with ROS detoxifying gold quantum clusters alleviates the functional decline in a mouse model of Friedreich ataxia

Although FRDA has no cure and few treatment options, there is research dedicated to finding an agent that can curb disease progression and address symptoms as neurobehavioral deficits, muscle endurance, and heart contractile dysfunctions. Because oxidative stress and mitochondrial dysfunctions are implicated in FRDA, this group demonstrated the systemic delivery of catalysts activity of gold cluster superstructures (Au8-pXs) to improve cell response to mitochondrial reactive oxygen species and thereby alleviate FRDA-related pathology in mesenchymal stem cells from patients with FRDA. The study also found that systemic injection of Au8-pXs ameliorated motor function and cardiac contractility of YG8sR mouse model that recapitulates the FRDA phenotype. These effects were associated to long-term improvement of mitochondrial functions and antioxidant cell responses. These events were related to an increased expression of frataxin, which was sustained by reduced autophagy. Overall, these results encourage further optimization of Au8-pXs in experimental clinical strategies for the treatment of FRDA.

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SS-31 efficacy in a mouse model of Friedreich ataxia by upregulation of frataxin expression

Previous studies have shown that SS-31, a mitochondrion-targeted peptide, is capable to upregulate the expression of FXN and improve the mitochondrial function in cells derived from FRDA patients. To further explore the potential of SS-31, the authors used the GAA expansion-based models including Y47 and YG8R mice, primary neurons and macrophages from the mice and cells derived from FRDA patients. After once-daily intraperitoneal injection of 1 mg/kg SS-31 for one month, the study showed significant improvement of motor function. The vacuolation in dorsal root ganglia, lesions in dentate nuclei and the lost thickness of myelin sheath of spinal cord were all repaired after SS-31 treatment. In addition, the hypertrophic cardiomyocytes and disarrayed abnormal Purkinje cells were dramatically reduced. Interestingly, the authors found that SS-31 treatment upregulated FXN expression not only at translational levels as observed in cell culture, but also at mRNA levels in vivo. Consequently, mitochondrial morphology and function were greatly improved in all tested tissues. Importantly, these data provided additional evidence that the maintenance of the therapeutic benefits needed continuous drug administration. Taken together, the findings have demonstrated the effectiveness of SS-31 treatment through the upregulation of FXN in vivo and offer guidance of the potential usage in clinical application for FRDA.

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ICAR Abstract Submission & Registration Opens September 9, 2021

Abstract Submission & Registration Opens September 9, 2021
The National Ataxia Foundation (NAF), the Friedreich’s Ataxia Research Alliance (FARA), and Ataxia UK are pleased to host the inaugural International Congress for Ataxia Research (ICAR) to be held at the Caribe Royale resort in Orlando, Florida, USA. Please save the date for March 15th – 18th, 2022.

ICAR 2022 will include 5 plenary sessions - Disease mechanisms, Cerebellar non-motor circuits and functions, Emerging and existing therapeutics, Late breaking, Interactive - and several breakout sessions, including ones on biomarkers and gene discovery. It will feature workshops with practical demonstrations and discussions on challenges of new and established technologies, and sessions and events specifically designed by and for junior investigators.

More details to follow at https://ataxiacongress.org/

Abstract Submission Schedule

September 9, 2021: Abstract Submission & Registration Open
November 8, 2021: Abstract Submission Deadline (11:59 pm ET)
December 15, 2021: Abstract Acceptance Announced
 
 

Reata Pharmaceuticals, Inc. Announces Second Quarter 2021 Financial Results and Provides an Update on Clinical Development Programs

PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage biopharmaceutical company, today announced financial results for the quarter ended June 30, 2021, and provided an update on the Company’s business operations and clinical development programs.

Recent Company Highlights

Omaveloxolone in Patients with Friedreich’s Ataxia (“FA”)

Based on a communication received from the U.S. Food and Drug Administration (“FDA”) regarding omaveloxolone for the treatment of FA, we withdrew our request for a Type C meeting and requested a pre-NDA meeting with the FDA. The pre-NDA meeting request has been granted, a pre-NDA meeting has been scheduled during the third quarter of this year, and we have submitted briefing materials for the meeting. We recently received a communication from the FDA requesting the estimated date of our New Drug Application (“NDA”) for its planning purposes. We plan to submit the NDA during the first quarter of 2022.

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Design Therapeutics Reports GeneTAC™ Portfolio Progress and Second Quarter 2021 Results

Preclinical Data Support Initiation of Clinical Development of Lead GeneTAC Program for Friedreich Ataxia in the First Half of 2022

Leadership Team Strengthened with Appointment of Elizabeth Gordon, Ph.D., as Senior Vice President of Regulatory Affairs

CARLSBAD, Calif., Aug. 09, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for degenerative genetic disorders, today reported recent progress with its portfolio of novel small molecule gene targeted chimeras (GeneTACsTM), as well as business highlights and second quarter 2021 financial results.

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