If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

"Moreover, the duration of follow-up is among the longest reported for a lentiviral vector-based gene therapy; 13 patients ...

As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene ...

For most parents, newborn screening tests are a barely perceptible safety net. But for the small minority whose babies get ...

As the May 19 legislative deadline approaches and we enter the final stretch of the 2025 session, the single most important ...

The committee’s termination came just weeks ahead of a meeting to discuss whether to add two new conditions: metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD). Both ...