Monday, March 9, 2026

Assessing Real-World Experiences on SKYCLARYS® (Omaveloxolone) (ARIES) in Patients With Friedreich Ataxia: An Observational Study

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MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026​. Poster Number: 292 T. Assessing Real-World Experiences on SKYCLARYS® (Omav...

Interim Analysis of mFARS Trajectories Across 5 Years in the MOXIe Extension: Impact of Omaveloxolone Initiation Timing in Friedreich Ataxia

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MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026, Poster Number: 297 T. Interim Analysis of mFARS Trajectories Across 5 Years...

Efficacy and Safety of a Novel Investigational AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich’s Ataxia

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MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026​. Poster Number: 299 T. Efficacy and Safety of a Novel Investigational AAV F...

Interim observations from a long-term open label study evaluating daily nomlabofusp administration in patients with Friedreich’s ataxia

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MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 419 O. Russell Clayton, DO, Larimar Therapeutics Inc., Erin ...

Unmet Needs of Individuals With Friedreich Ataxia Cardiomyopathy: Insights From the Lexeo Friedreich Ataxia Cardiac Advisory Council

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MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 293 T. Cardiomyopathy: Insights From the Lexeo Friedreich At...

Dexterity Outcomes in Friedreich Ataxia as Measured by mFARS and FA-ADL

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MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 294 T. Dexterity Outcomes in Friedreich Ataxia as Measured b...

Patient-reported experiences during long-term omaveloxolone treatment in Friedreich ataxia: survey design and planned qualitative analysis

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MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Patient-reported experiences during long-term omaveloxolone treatment in Fr...
Saturday, March 7, 2026

Health care, social, educational, and employment needs of individuals affected by rare diseases and their families in Catalonia, Spain

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Calmaestra-Carrillo, E., Vernet, R., Torrent-Farnell, J. et al. Health care, social, educational, and employment needs of individuals affect...

FDA reversals leave investors worrying about the fates of other experimental drugs

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Published Fri, Mar 6 2026. The FDA in the past year has denied or discouraged applications of at least eight new drugs, according to RTW Inv...
Thursday, February 26, 2026

Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA and Reiterates Planned BLA Submission in June 2026

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BALA CYNWYD, Pa., Feb. 24, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology comp...