Indication includes all ages and the full spectrum of disease severity –– First Japan product approval for PTC – WARREN, N.J., Dec. 22, 2025 ...
PTC Therapeutics, Inc. announced today that Sephiencetm (sepiapterin) has been approved by the Japanese Ministry of Health, Labor and Welfare (MHLW) for the treatment of children and adults living ...
The US HHS has added Duchenne muscular dystrophy and metachromatic leukodystrophy to newborn screening, citing benefits of early detection and access to FDA-approved treatments.
U.S. Health Secretary Robert F. Kennedy Jr. on Tuesday added two rare genetic disorders, Duchenne Muscular Dystrophy and Metachromatic Leukodystrophy, to the federal newborn screening list to enable ...
Nagpur: With the district sickle cell patient load rising to around 2,900, Indira Gandhi Govt Medical College and Hospital's Daga Hospital has made screening for the disease compulsory for all ...
Most international guidelines recommend lumbar punctures for all infants who have a fever at 28 days or younger to rule out bacterial meningitis. Clinical prediction tools may allow for more selective ...
An exam meant to determine whether a baby was born dead or alive is of “questionable value,” the National Association of ...
HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform ...
The finale also delivers two devastating reveals. First, Alexander learns that Rachel (Hannah Cruz), his ex-fiancée, ...
Children exposed to high levels of screen time before age 2 showed changes in brain development that were linked to slower ...