ASCLS eNewsBytes

• PTC wins FDA approval for first brain-delivered gene therapy Kebilidi November 18, 2024
  PTC Therapeutics' Kebilidi, the first U.S. gene therapy administered directly in the brain, has received FDA approval to treat the rare AADC deficiency. The minimally invasive procedure boosts the AADC enzyme and restores dopamine production.
• New stem cell data from space November 18, 2024
  Cedars-Sinai researchers have successfully introduced DNA into human induced pluripotent stem cells in space, a step closer to manufacturing stem cells in space. The experiments took place aboard Axiom Mission 2, Axiom Space's second astronaut mission to the International Space Station.
• Infection plus pollution equals increased Alzheimer's risk November 18, 2024
  A combination of a history of infections and exposure to traffic-related air pollution increases the risk for Alzheimer's disease by 177% in adults aged 60-75 years, according to data from around 100,000 individuals. The study found that vulnerability to infections plays a role in AD development, but the mechanism of action remains unclear.
• Pandemic linked to 16% rise in babies born with heart defects November 18, 2024
  Research from City St. George's, University of London shows that the number of babies born with congenital heart abnormalities increased by 16% during the first year of the pandemic, impacting one in 110 births globally. The study compared the number of babies born with Down's Syndrome.
• FDA approves Synda's Revuforj to treat leukaemia November 18, 2024
  The U.S. FDA has approved Syndax Pharmaceuticals' Revuforj for treating relapsed or refractory acute leukaemia with a lysine methyltransferase 2A gene translocation. Revuforj is now the first and only menin inhibitor approved for patients aged one year and older.