Milan, Italy, 12 September 2024 – Genespire, a biotechnology company developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases, today announces the appointment of ...
In the prioritized portfolio, the Company's rare disease candidates include therapies targeting two organic acidemias caused by deficient metabolic enzymes, methylmalonic acidemia (MMA ...
Sandra A. Banta-Wright, MN, RNC, NNP; Robert D. Steiner, MD Neonates with profound and prolonged hyperammonemia with coma due to urea cycle defect will have had a neurological insult to the brain ...