Sunday, August 23, 2020

Frataxin gene editing rescues Friedreich’s ataxia pathology in dorsal root ganglia organoid-derived sensory neurons

›
Pietro Giuseppe Mazzara, Sharon Muggeo, Mirko Luoni, Luca Massimino, Mattia Zaghi, Parisa Tajalli-Tehrani Valverde, Simone Brusco, Matteo Ja...
Saturday, August 22, 2020

Atypical structures of GAA/TTC trinucleotide repeats underlying Friedreich’s ataxia: DNA triplexes and RNA/DNA hybrids

›
Jiahui Zhang, Ashkan Fakharzadeh, Feng Pan, Christopher Roland, Celeste Sagui; Nucleic Acids Research, gkaa665, doi:10.1093/nar/gkaa665 Th...
Friday, August 21, 2020

Clinical presentation and survival of childhood hypertrophic cardiomyopathy: a retrospective study in United Kingdom

›
Gabrielle Norrish, Ella Field, Karen Mcleod, Maria Ilina, Graham Stuart, Vinay Bhole, Orhan Uzun, Elspeth Brown, Piers E F Daubeney, Amrit L...
Thursday, August 20, 2020

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

›
ClinicalTrials.gov Identifier: NCT04519567 Responsible Party: Larimar Therapeutics, Inc. First Posted: August 19, 2020 A Phase 1 Multiple...
Tuesday, August 18, 2020

Validation of low-cost system for gait assessment in children with ataxia

›
S. Summa, G. Tartarisco, M. Favetta, A. Buzachis, A. Romano, G.M. Bernava, A. Sancesario, G. Vasco, G. Pioggia, M. Petrarca, E. Castelli, E....
Monday, August 17, 2020

LARIMAR THERAPEUTICS : Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) share with twitter share with LinkedIn share with facebook

›
MarketScreener; 08/14/2020. CTI-1601 is currently being evaluated in Phase 1 clinical trials in patients with Friedreich's Ataxia. We h...

Larimar Therapeutics (LRMR) Investor Presentation - Slideshow

›
Seeking Alpha Aug. 13, 2020 6:05 PM ET | About: Larimar Therapeutics, Inc. (LRMR) About CTI-1601
Saturday, August 15, 2020

Identification of a Novel Oleic Acid Analog with Protective Effects in Multiple Cellular Models of Friedreich Ataxia

›
M Grazia Cotticelli, Roberto Forestieri, Shujuan Xia, Sipak Joyasawal, Taehee Lee, Kexin Xu, Amos B Smith Iii, Donna M Huryn, Robert B Wilso...
Thursday, August 13, 2020

Test–retest reliability of the Friedreich’s ataxia rating scale

›
Christian Rummey Theresa A. Zesiewicz Santiago Perez‐Lloret Jennifer M. Farmer Massimo Pandolfo David R. Lynch; Ann Clin Transl Neurol....
Wednesday, August 12, 2020

UPDATED: Reata suggests Friedreich's ataxia program could be delayed, sending stock plunging

›
August 10, 2020 05:07 PM EDT; Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial result...