The FDA has approved Kygevvi (doxecitine and doxribtimine) for the treatment of TK2d in adults and pediatric patients with an age of symptom onset on or before 12 years.
We have written about the promise of gene therapy to correct genetic disease many times. An exciting development happened ...
Although heart cells and skin cells contain identical instructions for creating proteins encoded in their DNA, they're able ...
UCB has scored an FDA approval for ultra-rare disease treatment Kygevvi, continuing the positive momentum for the Belgian ...
The approval was based on data from a Phase II trial, two retrospective chart review studies and an expanded access programme ...
Researchers at Memorial Sloan Kettering Cancer Center (MSK) have developed some of the first robust laboratory models of a ...
Edited by Lora Hooper, The University of Texas Southwestern Medical Center, Dallas, TX; received March 18, 2025; accepted August 4, 2025 ...
Within each cell, there is a constant struggle between life-sustaining molecules and molecules that could hasten its demise. For the first time, scientists have created a molecule that can identify ...
VEXAS syndrome is an a confounding adult-onset inflammatory disease first described in 2020, which affects 1 in 4,000 men ...
A study led by UCLA Health Jonsson Comprehensive Cancer Center investigators reveals how melanoma, the deadliest form of skin cancer, evolves to resist immunotherapy and identifies a potential ...
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