For patients with severe Leukocyte Adhesion Deficiency Type I (LAD-I), treatment options have been limited. A new gene therapy may offer hope through the FDA’s accelerated approval pathway. https://t.co/MosJOFGOuY
In a landmark scientific feat published in @NEJM, a @ChildrensPhila & @PennMedicine team treated the first patient with personalized #genetherapy, designed uniquely for newborn KJ, who was diagnosed with a rare #ureacycledisorder at birth. Learn more: https://t.co/aUS67Sncdy
A new study in @NEJM reports that an investigational gene therapy restored immune function in 9 children with LAD-I, a rare & life-threatening immune disorder. Those children are now living symptom-free 2 years after treatment in clinical trials co-led by @Kohn_Lab@UCLA.
The 2024 IUIS Inborn Errors of Immunity (IEI) Classification has arrived! Explore our latest insights now at https://t.co/IqbLhAGPTq. Find the report under “Annual 2024 Report”!
I’m honored to have performed tonight, for the Paris 2024 Opening Ceremony, and so full of joy to be back in one of my very favorite cities! Most of all, I’m so happy to be celebrating these amazing athletes, with all their stories of sacrifice and determination, pain and perseverance. All of you have been so focused on your dream, and whether or not you take home a medal, I hope that being here means that it has come true for you! You should all be so proud, we know how hard you have worked to be the best of the best. Stay focused, keep going, my heart is with you! - Celine xx…
Photo: Laura Gilli
We're celebrating the International Day of Women and Girls in Science!
Let's look back on the life of Marie Skłodowska Curie: a Nobel Prize laureate who dedicated her life to science and became one of the world's greatest scientists.
#WomenInScience#NobelPrize
Acid-suppressive medication and antimicrobial use in infancy associated with food allergy and anaphylaxis according to new research being presented at the 2024 AAAAI Annual Meeting. #AAAAI24#FoodAllergy
https://t.co/aOEH7rbaNu
Remarkable and entertaining introduction to the field of IEI by @HsiehElena@alexbolze & Joseph Hernandez:
Inborn errors of immunity illuminate mechanisms of human immunology and pave the road to precision medicine https://t.co/hIZL6aNtxq
Additionally, one of these therapies is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
Today we approved two milestone treatments - the first cell-based gene therapies to treat sickle cell disease in patients 12 years and older. https://t.co/az8lDdRyz0