Levacetylleucine (Aqneursa) improved functioning in a phase III crossover trial of adults and children with ataxia-telangiectasia. Two patients had three treatment-emergent adverse events; none were ...

(BHVN) closed 7% higher on Wednesday on the heels of hitting enrollment milestones in two high-priority clinical programs, ...

Incubated by Houston-based Fannin Innovation Studio, the 11h drug created in Nebraska will move toward phase 1 clinical ...

Only successful pivotal Phase III trial in Ataxia-Telangiectasia (A-T), a rare, progressive neurodegenerative disease with no approved treatments Phase III trial successfully achieved its primary ...

IntraBio Inc. today announced that results from its pivotal Phase III clinical trial evaluating levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T) have been published in The Lancet ...

Prime Minister Sir Kier Starmer was challenged to help Jake in the House of Commons ...

Larimar submitted the module of a rolling biologics license application to the U.S. Food and Drug Administration for the ...

Published in the open-access journal Ferroptosis and Oxidative Stress, the review by Giovanni Cravin and Giorgio Cozza from ...

Niagen Bioscience, Inc. (NASDAQ: NAGE), the global authority on NAD+ science, today announced the formal launch of the first drug candidate of NAD Pharmaceuticals Corp., its wholly owned subsidiary ...

Eimear McAndrew was diagnosed with early-onset Spinocerebellar Ataxia 7 (SCA7) at 27 years old, which is an aggressive ...

Larimar Therapeutics Inc. LRMR said Monday it has submitted the first module of a rolling Biologics License Application to ...

SUNRISE-FA 2 study parameters include LVMI primary endpoint, 6-month topline efficacy analysis, inclusion criteria focused on abnormal baseline ...