Tag: Gene Therapy
Gene Therapy Effective in Patients With Recessive Dystrophic Epidermolysis Bullosa
One-time surgical application of prademagene zamikeracel improves wound healing and pain
FDA Approves Zevaskyn for Rare, Genetic Skin Disorder
Zevaskyn is the first and only autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa
Sarepta’s Gene Therapy Under Scrutiny After Patient Death
FDA Approves Kebilidi for Aromatic L-Amino Acid Decarboxylase Deficiency
Approval represents first gene therapy for treatment of AADC deficiency
Lentiviral Gene Therapy Beneficial for Early Cerebral Adrenoleukodystrophy
No major functional disabilities observed at month 24; 81 percent of patients had no major functional disabilities at median of six years
Hematologic Cancer Develops in Some Patients Receiving Eli-Cel
Hematologic cancer developed in seven of 67 patients with cerebral adrenoleukodystrophy receiving eli-cel gene therapy
Fidanacogene Elaparvovec Superior to Prophylaxis for Hemophilia B
Adeno-associated virus gene-therapy vector for hemophilia B superior for reducing annualized bleeding rate
Bilateral Gene Therapy Safely Restores Hearing in Children Born Deaf
Bilateral hearing restoration seen for all five patients; speech perception and capability of sound source localization restored
Breakthrough Gene Therapy Enables Infant Born Deaf to Hear
Child's hearing and speech are currently on track, parents and doctors say
First Gene Therapy for Children With Metachromatic Leukodystrophy Approved by FDA
Approval is for a single-dose gene therapy infusion
