Medscape12d
Cognitive strengths and weaknesses in children and adolescents homozygous for the galactosemia Q188R mutation: a descriptive study.
Children and adolescents (n = 25) with galactosemia homozygous for the common Q188R mutation (substitution of glutamine codon 188 with arginine) were group matched for sex and age with healthy ...
Hosted on MSN2mon
APLT Stock Plunges 77% After FDA Issues CRL for Galactosemia Drug NDA
In the phase III ACTION-Galactosemia Kids study in children with galactosemia aged 2-17 years, govorestat improved daily activities, behavior, cognition, fine motor skills and tremor. It also ...
JD Supra1mon
Complete Response Letter, Warning Letter and Shareholder Lawsuit Follow FDA Data Integrity Findings
Applied Therapeutics relied on data from its phase 3 ACTION-Galactosemia Kids trial in children ages 2 – 17 with galactosemia and its phase 1/2 ACTION-Galactosemia trial in adult patients with ...
Medindia6y
Medications and Drugs for Treatment of Galactosemia
Looking for reliable medications to treat 'Galactosemia'? This page offers a detailed resource for the most up-to-date treatment options, including both generic and brand-name medications.
Nature11mon
CORRELATION OF OVARIAN FUNCTION WITH GALACTOSE-l-PHOSPHATE URIDYL TRANSPERASE LEVELS IN GALACTOSEMIA
Galactosemia (G) is due to either partial or complete deficiency of the enzyme galactose-1-phosphate uridyl transferase (Ts). In females with G, there is a high incidence of premature ovarian ...