Gene Therapy
Q&A: ‘An exciting breakthrough’: Gene transfer therapy promising in Danon disease
Danon disease is a rare but serious cardiomyopathy that drastically reduces life expectancy.
European Medicines Agency recommends ‘landmark’ gene therapy for epidermolysis bullosa
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended the approval of Vyjuvek for the treatment of wounds in patients with dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.
VIDEO: MCO-010 demonstrates vision improvements, safety in retinitis pigmentosa
KOLOA, Hawaii — In this Healio Video Perspective from Retina 2025, Allen C. Ho, MD, discusses the growing promise of optogenetic therapy in treating patients with retinal disease.
Gene therapy slows progression of spinal muscular atrophy type 2
Topline results from a phase 3 clinical trial showed an investigational gene therapy for young people with spinal muscular atrophy type 2 increased mobility and slowed disease progression, according to a press release from Novartis.
BLOG: Gene therapies for children with sickle cell disease
An increasing number of gene therapies correct genetic disorders, including hemoglobinopathies.
Novel cell therapy ‘potentially life changing’ for people with sickle cell disease
SAN DIEGO — An investigational cell therapy reduced sickle hemoglobin and increased fetal and total hemoglobin for certain patients with sickle cell disease, according to results presented at ASH Annual Meeting and Exposition.
ASH presents honorific awards to ‘visionary’ hematologists
Several hematologists will be honored during this year’s ASH Annual Meeting.
ASH sickle cell disease research priorities address pain, organ dysfunction, drug development
American Society of Hematology has issued new sickle cell disease research priorities, highlighting seven key focus areas for maximum impact and benefit to patients.
FDA investigating hematologic cancer risk in kids treated with Skysona for brain disease
The FDA announced it is investigating serious health risks associated with administration of an autologous hematopoietic stem cell gene therapy for children with early active cerebral adrenoleukodystrophy, or CALD.
FDA approves novel gene therapy for rare neurometabolic disorder
The FDA has approved an adeno-associated virus vector-based gene therapy to treat both adult and pediatric patients with aromatic L-amino acid decarboxylase deficiency.