The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have decreased AADC activity that causes severe disability, including gross motor ...

An assessment of the natural history cohort showed none of the 43 untreated patients had documented motor milestone achievement at last follow-up at a median age of 7.2 years (range, 2-19 years).

Canadian physicians should be aware of the higher risk of 1-α-hydroxylase deficiency in the French-Canadian populations of Quebec and the Maritime provinces. Twin 12-month-old girls underwent ...

After 48 weeks of treatment, 12 out of the 13 patients had completed motor milestone assessments. Final results found that Kebillidi demonstrated gross motor function improvement in eight of 12 ...

FDA grants accelerated approval to PTC Therapeutics' Kebilidi, the first brain-administered gene therapy in the U.S. for AADC deficiency. Clinical trials show Kebilidi improved motor function in 8 ...

The effect of subclinical infantile thiamine deficiency on motor function in preschool children. Maternal & Child Nutrition , 2017; e12397 DOI: 10.1111/mcn.12397 Cite This Page : ...

"We've proven that B1 deficiency in infancy has long-term implications on gross and fine motor function and balance skills in childhood," said Prof. Fattal-Valevski.

July 12 (UPI) --Research suggests that a vitamin D deficiency in pregnancy has negative effects on social development, motor skills in preschool age children.Researchers from the University of ...

At baseline, trial participants had no gross motor function -- the most severe presentation of AADC deficiency -- and decreased AADC activity in plasma. Motor assessments were completed for 12 ...

A new Tel Aviv University study found that infantile vitamin B1 (thiamine) deficiency severely affected the motor function of preschoolers who were fed faulty formula in the first year of their lives.