Adrenoleukodystrophy
JAMA 294:3131-3134, Moser,H.W.,et al, 2005
Long-Term Effects of Antidarsagene Autotemcel for Metachromatic Leukodystrophy
NEJM 392:1609-1620, Fumagalli,F.,et al, 2025
Newborn Screening and Presymptomatic Treatment of Metachromatic Leukodystrophy
NEJM 391:1256-1258, Laugwitz,L.,et al, 2024
Cerebrovascular Events in Patients Undergoing Transcatheter Aortic Valve Replacement:A Review
Stroke 55:2754-2764, Patel,K.P.,et al, 2024
"Im fine; Im just waiting for my disease" The New and Growing Class of Presymptomatic Patients
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