After experiencing setbacks, PTC reported positive results for vatiquinone, its selective inhibitor of 15-Lipoxygenase (15-LO ...

Larimar has undertaken an ongoing open-label extension study of nomlabofusp with the first enrolled patient being dosed in ...

Bill Nye the Science Guy is temporarily rebranding. For now, he’s “Bill Nye the Ataxia Advocate Guy,” as he aims to bring awareness to a neurological disease that has affected his family for ...

Sami Corwin has given his Buy rating due to a combination of factors surrounding PTC Therapeutics’ promising drug, vatiquinone, for the treatment of Friedreich ataxia (FA). The latest results ...

"That doctor put the MRI of my head on the screen and immediately said I have ataxia," said Pepper. "I said, 'What's that?'" Ataxia is a condition typically resulting from damage to the cerebellum.

BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has released its financial results for the third quarter of 2024, ...

The ATM gene (mutated in the disease ataxia telangiectasia ... functions upstream of p53 in the IR-induced signal transduction pathway 5. Although much information on the regulation of p53 ...

AT = ataxia–telangiectasia ... The biological effects of p53 pathway variants at the molecular level in primary cells or in vivo still need to be determined. The design of genetically engineered ...

This condition eventually leads to ataxia, speech problems ... and I believe it has a feasible pathway towards regulatory approval. The company also has enough resources to fund its research ...

(NASDAQ:GRCE) with a valuation of $12.50 per share. This value is based on our estimates for the successful development and commercialization of GTX-104 in patients being treated for aneurysmal ...

Nomlabofusp program update expected mid-December to include available safety, pharmacokinetic (PK) and frataxin data, as well as available ...

The results in Alzheimer's come a few months after Lexeo also reported encouraging data with its gene therapy for ...