We thank Michèl Willemsen and colleagues for their interest in the ATTeST trial of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia. 1 The two doses studied in ...

aDepartment of Pediatrics (Pediatric Neurology), Amalia Children's Hospital, Radboud University Medical Center, Nijmegen 6500, Netherlands ...

Macular telangiectasia, sometimes referred to as idiopathic juxtafoveal macular telangiectasia, is a disease that affects the part of the eye called the macula, causing degradation or loss of central ...

19, 2025. "We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich's ataxia," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics.