Larimar Therapeutics, Inc. (NASDAQ:LRMR) released initial data from the ongoing long-term OLE study evaluating daily ...

Early attempts to treat FRDA with antioxidants, before the FXN gene was discovered, were prompted by a generic assumption that oxidative stress contributes to neurodegeneration, as well as by the ...

After Natalie Rex was diagnosed with Friedreich’s ataxia -- a rare genetic and usually fatal neurological disease -- her twin ...

The disorder is caused by an erroneous long chain of repeating DNA bases in the FXN gene, which prevents the cell’s transcriptional machinery from reading the entire gene and producing the ...

inherited neuromuscular disease caused by mutations in a gene called FXN that leads to impaired muscle coordination, as well as problems with the heart and central nervous system. Earlier this ...

In the assessment of 12-month price targets, analysts unveil insights for Voyager Therapeutics, presenting an average target ...

Its pipeline of programs include, Anti-tau Antibody (VY-TAU01), SOD1 Silencing Gene Therapy (siRNA), FXN Gene Therapy among others. Market Capitalization: Exceeding industry standards, the company ...

Treatment with nomlabofusp modified gene expression and lipid profiles in addition to increasing frataxin – FXN – levels in study participants with Friedreich’s ataxia. Modeling and ...

As FA is genetic, a diagnosis is made by testing for a mutation in the gene FXN, which is responsible for causing the disease. Stiff Person Syndrome Patients Share What It’s Like To Live With ...

inherited neuromuscular disease caused by mutations in a gene called FXN that leads to impaired muscle coordination, as well as problems with the heart and central nervous system. Earlier this ...