GEN4 天
Rare Genetic Disorder Helps Explain Link Between Cancer Immunotherapy and Increased Infections
Studying patients with PD-1 or PD-L1 deficiency may help inform new approaches to fine-tuning immunotherapies to maximise ...
WebMD13 天
First Brain-Injected Gene Therapy Approved by FDA
The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called ...
Medpage Today on MSN18 天
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
Nature10 天
Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype ...
The CNS-specific phenotype of Mecp2 deficiency is unexpected considering that the gene is expressed ubiquitously and thought to act as a general repressor of transcription. It remains a major ...
Science Daily5 天
Gene Therapy News
Sep. 9, 2024 — Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs ...
World's youngest patient receives groundbreaking gene therapy in Cincinnati
A groundbreaking gene therapy treatment at Cincinnati Children's Hospital has given a 3-year-old boy named Sriansh a new ...
GlobalData on MSN18 天
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) ...
PD-1 deficiency reveals the impact of checkpoint inhibition on B cell function
A multinational collaboration co-led by the Garvan Institute of Medical Research has uncovered a potential explanation for why some cancer patients receiving a type of immunotherapy called checkpoint ...
Monthly Prescribing Reference17 天
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
Too Old to Operate4 天
FDA Approves Kebilidi for Aromatic L-Amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration has approved Kebilidi (eladocagene exuparvovec-tneq) for the treatment of adult and ...