Initial Data from the Ongoing, Blinded Phase 1 Single-Ascending Dose Trial Has Demonstrated Favorable Safety and Pharmacokinetics IND Application Submitted to Expand RESTORE-FA Trial to U.S. Sites; ...

Researchers have developed a gene editing strategy that can repair the genetic problems that lead to Huntington's disease and Friedreich's ataxia. | Genetics And Genomics ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

After a rocky start to the spring, Wall Street came roaring back in May. President Trump’s softened tariff stance reignited ...

Treatment with a 150 mg daily oral dose of omaveloxolone over 48 weeks in the study resulted in a significant improvement in the symptoms of Friedreich’s ataxia – measured using the mFARS ...

Its lead product candidate is CTI-1601, which is in Phase 2 OLE clinical trial for the treatment of Friedreich’s ataxia, a rare, progressive and fatal genetic disease. The company is based in ...

A Columbia woman is putting a spotlight on a rare, degenerative disease, which targets the nervous system and often impairs ...

Currently, there is no FDA-approved treatment for the devastating neurodegenerative ... nomlabofusp protein replacement therapy for Friedreich’s ataxia (FA), a devastating and progressive ...

Owen Donoghue, 44, took on the challenge to help eight-year-old Craigshill girl Ella-Rose Kennedy who has Friedreich ataxia – ...

Early research shows that editing expanding trinucleotide repeats halts the lengthening process that causes neurological disease.

UK drivers have been told they could be slapped with a fine by the Driver and Vehicle Licensing Agency if not following certain rules ...

Figure 5: Sca10 is widely expressed in brain regions that are anatomical substrates for ataxia and epilepsy. We extracted DNA from blood samples of all participating family members after obtaining ...