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Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C

Bremova-Ertl, Tatiana ; Ramaswami, Uma ; Brands, Marion ; Foltan, Tomas ; Gautschi, Matthias ; Gissen, Paul ; Gowing, Francesca ; Hahn, Andreas ; Jones, Simon ; Kay, Richard ; Kolnikova, Miriam ; Arash-Kaps, Laila ; Marquardt, Thorsten ; Mengel, Eugen ; Park, Julien H. ; Reichmannová, Stella ; Schneider, Susanne A. ; Sivananthan, Siyamini ; Walterfang, Mark ; Wibawa, Pierre ; Strupp, Michael ; Martakis, Kyriakos

The New England journal of medicine, 2024-02, Vol.390 (5), p.421-431 [Periódico revisado por pares]

United States: Massachusetts Medical Society

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Título:
Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C
Autor: Bremova-Ertl, Tatiana ; Ramaswami, Uma ; Brands, Marion ; Foltan, Tomas ; Gautschi, Matthias ; Gissen, Paul ; Gowing, Francesca ; Hahn, Andreas ; Jones, Simon ; Kay, Richard ; Kolnikova, Miriam ; Arash-Kaps, Laila ; Marquardt, Thorsten ; Mengel, Eugen ; Park, Julien H. ; Reichmannová, Stella ; Schneider, Susanne A. ; Sivananthan, Siyamini ; Walterfang, Mark ; Wibawa, Pierre ; Strupp, Michael ; Martakis, Kyriakos
Assuntos: Adolescent ; Adult ; Adverse events ; Aged ; Allergy ; Ataxia ; Central Nervous System Agents - administration & dosage ; Central Nervous System Agents - therapeutic use ; Child ; Child, Preschool ; Childhood Diseases ; Clinical trials ; Cross-Over Studies ; Data Collection ; Double-Blind Method ; Drug dosages ; FDA approval ; Gait ; Genetics ; Humans ; Immunology ; Inflammatory Disease ; Leucine ; Leucine - analogs & derivatives ; Leucine - therapeutic use ; Long-term effects ; Middle Aged ; Neurology ; Neuroscience ; Neurosurgery ; Neurosurgery General ; Niemann-Pick disease ; Niemann-Pick Disease, Type C - complications ; Niemann-Pick Disease, Type C - diagnosis ; Niemann-Pick Disease, Type C - drug therapy ; Niemann-Pick Disease, Type C - genetics ; Oral administration ; Patients ; Pediatrics ; Pediatrics General ; Placebos ; Treatment Outcome ; Young Adult
É parte de: The New England journal of medicine, 2024-02, Vol.390 (5), p.421-431
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Descrição: In Niemann–Pick disease type C, a rare lysosomal storage disorder, treatment with N -acetyl- l -leucine, which may ameliorate lysosomal and metabolic dysfunction, resulted in better neurologic status than placebo.
Editor: United States: Massachusetts Medical Society
Idioma: Inglês

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Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C

United States: Massachusetts Medical Society

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