Results 91 to 100 of about 3,578 (140)

Mitochondrial iron overload is associated with lysosomal dysfunction-mediated mitophagy impairment in the heart of Friedreich's ataxia. [PDF]

Mitochondrion
Jee E, Medha M, Baek H, Kim J, Kim Y.
europepmc   +1 more source

Characterizing Population Pharmacokinetics of Vatiquinone in Healthy Volunteers and Patients with Friedreich's Ataxia. [PDF]

Pharmaceuticals (Basel)
Hu Y, Gao L, Lee L, Cherry JJ, Kong R.
europepmc   +1 more source

Early experience on omaveloxolone in adult patients with Friedreich's ataxia: a real-world observational study. [PDF]

J Neurol
Lima SM, Caltagirone M, Messina C, Quartetti U, Rini N, D'Amico F, Brighina F, Di Stefano V.   +7 more
europepmc   +1 more source

Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia. [PDF]

Mol Ther
Tang H, Gupte S, Xu E, Calabro KR, Friend H, Crosson SM, Fajardo D, Kostamo Z, Zhang H, Peterson JJ, Lin F, Kozmik Z, Lutz CM, Boye SL, Boye SE.   +14 more
europepmc   +1 more source

Generation of Friedreich's ataxia induced pluripotent stem cells carrying the FXN c.165 + 5G>C splicing mutation. [PDF]

Stem Cell Res
Yameogo P, Gerhart BJ, Sentmanat MF, Neilson A, Cui X, Verma M, Lynch DR, Napierala JS, Napierala M.   +8 more
europepmc   +1 more source

Pharmacokinetics and Pharmacodynamics of Nomlabofusp in Non-clinical Studies of Friedreich's Ataxia. [PDF]

AAPS J
De Toni F, Ragaglia V, Schecter D, Miller AS, Gonzalez E, Wagner EJ, Xu X, Payne RM, Mess JN, Baile MG, Clements-Egan A, Shankar G.   +11 more
europepmc   +1 more source

Safety and efficacy of individualised exercise and NAD⁺ precursor supplementation in patients with Friedreich's ataxia in the USA: a single-centre, 2 × 2 factorial, randomised controlled trial. [PDF]

Lancet Neurol
Lin KY, Bucha A, McSweeney K, Wade KL, Karaj A, Tamaroff J, O'Malley S, Chung NM, Cilenti NA, Wanner J, Adzika GK, Mesaros C, Blair IA, Rojsajjakul T, Serai S, Farmer J, Bryant K, Lu Y, Harhay MO, Weber DR, Paridon SM, Seifert EL, Putt ME, Zamani P, Baur JA, Lynch DR, McCormack SE.   +26 more
europepmc   +1 more source

Muscle Endurance Training in a Person with Friedreich's Ataxia. [PDF]

Muscles
McGarrell NT, Green ME, McCully KK.
europepmc   +1 more source

Dysregulation of sphingolipid-metabolizing enzymes in Friedreich's ataxia: <i>In vitro</i> and <i>in vivo</i> insights into therapeutic targeting. [PDF]

iScience
Ramchunder Z, Kalef-Ezra E, Suleman S, Edzeamey FJ, Szunyogh S, Gittins O, Castro Mena N, Wade-Martins R, Valle A, Pourzand C, Anjomani Virmouni S.   +10 more
europepmc   +1 more source

Editorial: The mechanistic investigation and emerging therapies for Friedreich's ataxia. [PDF]

Front Pharmacol
Dong Y, Chandran V, Soragni E, Lynch DR.
europepmc   +1 more source