The American Journal of Managed Care

Friedreich Ataxia: Addressing Treatment Objectives and Comorbidities

Treatment goals for Friedreich ataxia include symptom management, slowing disease progression, and enhancing quality of life through multidisciplinary care. Supportive therapies address comorbidities ...
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Biogen's Skyclarys Approved In Europe For Friedreich's Ataxia Treatment

(RTTNews) - Biogen Inc. (BIIB) said that the European Commission has authorized Skyclarys (omaveloxolone) for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.

Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations - - Dosing of the first participant ...
Healio

VIDEO: First approved ataxia treatment reduces cell stress, improves antioxidants

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Medical Xpress

Genetic modifier of Friedreich's ataxia points toward treatment for devastating disorder

Friedreich's ataxia (FA) is a rare but devastating genetic disorder. Those with the condition are often diagnosed between 5 and 15 years of age and live only into their 30s or 40s. There is no widely ...
The American Journal of Managed Care

Managing Advanced Stages of Friedreich’s Ataxia: Challenges and Emerging Therapeutic Avenues

A health care provider outlines treatment objectives and supportive interventions for managing Friedreich ataxia, addressing associated comorbidities and symptoms in clinical settings. August 7th ...

Solid Biosciences Doses First Participant in First-in-Class Phase 1b FALCON Trial Evaluating SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

Initial data from the Phase 1b FALCON trial expected in H2 2026, subject to participant enrollmentCHARLESTOWN, Mass., Jan. 12 ...
Business Wire

Reata Pharmaceuticals Announces FDA Approval of SKYCLARYS™ (Omavaloxolone), the First and Only Drug Indicated for Patients with Friedreich’s Ataxia

“Friedreich's ataxia is a debilitating neuromuscular disease that progressively robs patients of their mobility and independence,” said Susan Perlman, MD, Clinical Professor, Department of Neurology, ...
Healio

FDA requests more studies before approving vatiquinone for Friedreich’s ataxia

Please provide your email address to receive an email when new articles are posted on . Vatiquinone inhibits 15-Lipoxygenase, which regulates the pathways that Friedreich’s ataxia disrupts. PTC ...
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Solid Biosciences' SGT-212 Gene Therapy for Friedreich's Ataxia Receives FDA IND Clearance, Phase 1b Trial Set for 2025

Solid Biosciences announces FDA clearance for SGT-212, targeting Friedreich’s ataxia with dual gene therapy administration routes, initiating Phase 1b trial in 2025. Solid Biosciences Inc. announced ...
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PTC Therapeutics Announces Positive Results from Long-Term Treatment Studies and Updates on Regulatory Progress for Vatiquinone Friedreich Ataxia Program

- Following FDA pre-NDA feedback, submission on track for December 2024 - WARREN, N.J., Oct. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (PTCT) provided today several positive updates on the ...
Business Wire

ChromaDex Receives Exclusive U.S. FDA Orphan Drug Designation (ODD) and Rare Pediatric (RPD) Disease Designation for Nicotinamide Riboside Chloride (NRC) for the Treatment of ...

LOS ANGELES--(BUSINESS WIRE)--ChromaDex Corp. (NASDAQ:CDXC), the global authority on nicotinamide adenine dinucleotide (NAD +) and healthy aging research, today announced that the U.S. Food & Drug ...