by Madaline Spencer | Mar 12, 2026
The US Food and Drug Administration (FDA) has accepted DF-003 into the FDA Rare Disease Evidence Principles Process (RDEP) for the potential treatment of ROSAH syndrome. ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache) syndrome is a...
by Madaline Spencer and James Radke, PhD | Mar 12, 2026
Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses the first-in-human experience of autologous hematopoietic stem cell gene therapy (HSCGT) using a novel ex-vivo lentiviral (LV) platform to correct mucopolysaccharidosis II...
by Madaline Spencer and James Radke, PhD | Mar 11, 2026
Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the challenges and unmet needs of female patients with Fabry disease. Fabry disease is a type of lysosomal storage disease characterized by deficient alpha-galactosidase...
by Madaline Spencer and Joe Haddad | Mar 10, 2026
The US Food and Drug Administration (FDA) has approved Tecvayli (teclistamab-cqyv) plus Darzalex Faspro (daratumumab and hyaluronidase-fihj) for the treatment of adults with relapsed or refractory multiple myeloma (R/R MM) who have received at least one prior line of...
by Madaline Spencer | Mar 9, 2026
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp for the treatment of patients with Friedreich’s ataxia. Friedreich’s ataxia is a progressive, inherited condition that affects the nervous system and causes...
