Mutation
Vanzacaftor, tezacaftor, deutivacaftor similar to current cystic fibrosis standard of care
Among individuals aged at least 12 years with cystic fibrosis, receipt of vanzacaftor/tezacaftor/deutivacaftor was similar or better than Trikafta depending on the endpoint, according to results published in The Lancet Respiratory Medicine.
FDA approves Alyftrek for cystic fibrosis treatment
The FDA has approved Alyftrek for patients aged 6 years and older with cystic fibrosis with certain mutations, according to a press release from Vertex Pharmaceuticals.
Non-Small Cell Lung Cancer
Treatment Options and Emerging Therapies
Precision and Immune-Based Therapies
VIDEO: SOD1 gene shows ‘great promise’ in ALS treatment
In this video, Alokkumar Jha, PhD, MSc, BE, spoke with Healio about treating patients with ALS with a mutation in the superoxide dismutase 1, or SOD1 gene.
Q&A: Finding a treatment for rare cystic fibrosis splicing mutation
Although inhaled antisense oligonucleotide treatment is safe, its efficacy needs to be determined in patients with the cystic fibrosis splicing mutation 3849+10kb C-to-T, according to a press release from the Cystic Fibrosis Foundation.
FDA expands approval of Elevidys to children aged 4 years and older with DMD gene mutation
The FDA has announced approval of a label expansion for Elevidys to include patients with Duchenne muscular dystrophy aged 4 years and older with a confirmed mutation in the DMD gene.
VIDEO: Research focuses on non-coding mutations in North Carolina macular dystrophy
WAILEA, Hawaii — In this Healio Video Perspective from Retina 2024, Kent W. Small, MD, discusses research exploring non-coding mutations in North Carolina macular dystrophy.
VIDEO: Progression of pre-MDS conditions into MDS AML
Healio spoke with Akriti Jain, MD, about the pre-MDS conditions that have the potential to progress into MDS if left untreated.