News

Early Data Support Gene-editing Platform’s Ability to Fix FA Deficits

Prime Medicine’s gene-editing technology — called Prime Editing — successfully corrected the genetic deficits associated with Friedreich’s ataxia (FA) and restored nerve cell projections in preclinical studies using patient cells, the company has announced. The company also announced positive updates from a number of its 18 other developmental programs…

Spinal Cord Abnormalities Correlate With Disease Severity in FA

People with Friedreich’s ataxia show marked abnormalities in the structure of the upper spinal cord, some of which are correlated with measures of disease duration and severity, a study reports. “These results provide new insights about [the development of Friedreich’s ataxia] and indicate that spinal cord MRI may be…

$4.8M Grant Supports Work Into Gene Editing Treatment for FA

A $4.8 million grant from the California Institute for Regenerative Medicine (CIRM) will support a research project hoping to move a one-time gene editing treatment for Friedreich’s ataxia closer to clinical trials. “If successful, this approach could prevent the neurologic and cardiac complications in [Friedreich’s ataxia] and address the pressing…

Diabetes Linked to Worse COVID-19 Outcomes in FA Patients

People with Friedreich’s ataxia (FA) who also have diabetes, a condition that causes their blood glucose (sugar) level to become too high, are more likely than those who don’t have diabetes to have worse outcomes from COVID-19, according to a single-center study. Having diabetes increased both the chance of…

Larimar’s CTI-1601 Phase 2 Trial Now Enrolling in US

A four-week Phase 2 clinical trial that’ll be testing CTI-1601, Larimar Therapeutics’ experimental therapy for Friedreich’s ataxia (FA), is now enrolling in the U.S., the company announced in a press release. Patient recruitment began shortly after the U.S. Food and Drug Administration (FDA) lifted its full hold…

A Conversation With Rare Disease Advocates