Acetyl-leucine slows disease progression in lysosomal storage disorders

E Kaya, DA Smith, C Smith, L Morris… - Brain …, 2021 - academic.oup.com
… Another major finding of the current study was that ADLL and ALL (but not ADL) slowed
disease progression when treatment was initiated before symptom onset, consistent with a …

Acetyl-leucine slows disease progression in lysosomal storage disorders

E Kaya, DA Smith, C Smith, L Morris, T Bremova-Ertl… - sussex.figshare.com
… Another major finding of the current study was that ADLL and ALL (but not ADL) slowed
disease progression when treatment was initiated before symptom onset, consistent with a …

[PDF][PDF] Acetyl-leucine slows disease progression in lysosomal storage disorders Short title: Acetyl-leucine for lysosomal diseases

E Kaya, DA Smith, C Smith, L Morris, T Bremova-Ertl… - 2020 - researchgate.net
… and ALL (but not ADL) slowed disease progression when treatment was initiated before
symptom … as this actively contributes to disease progression and reducing inflammation using …

[HTML][HTML] Beneficial effects of acetyl-DL-leucine (ADLL) in a mouse model of Sandhoff disease

E Kaya, DA Smith, C Smith, B Boland, M Strupp… - Journal of clinical …, 2020 - mdpi.com
disease is a rare neurodegenerative lysosomal storage disease associated with the storage
… but significantly extended lifespan and significantly slowed disease progression in Hexb -/- …

[HTML][HTML] N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study

T Fields, T M. Bremova, I Billington, GC Churchill… - Trials, 2023 - Springer
… The modified amino acid N-acetyl-leucine has been … , as well as slowing disease progression
and prolonging survival (… treatment of lysosomal storage disorders and neurodegenerative …

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

T Bremova-Ertl, U Ramaswami, M Brands… - … England Journal of …, 2024 - Mass Medical Soc
… The disease manifests with systemic, psychiatric, and … disease type C is currently limited to
slowing the progression of … for glycosphingolipid lysosomal storage disorders. Miglustat has …

[HTML][HTML] Efficacy and safety of N-acetyl-l-leucine in Niemann–Pick disease type C

T Bremova-Ertl, J Claassen, T Foltan… - Journal of …, 2022 - Springer
… , slowed disease progression, and prolonged survival—… , disease-modifying effects of
acetyl-leucine were found in an analogous animal model of another lysosomal storage disease, …

Acetyl-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study

T Fields, T Bremova-Ertl, I Billington, G Churchill… - 2022 - researchsquare.com
… The modi ed amino acid N-acetyl-leucine has been … , as well as slowing disease progression
and prolonging survival (… treatment of lysosomal storage disorders and neurodegenerative …

[HTML][HTML] N-acetyl-L-leucine: a promising treatment option for traumatic brain injury

C Sarkar, MM Lipinski - Neural Regeneration Research, 2022 - journals.lww.com
… it is also a major risk factor for development of neurodegenerative diseases like Alzheimer’s
… effect in lysosomal storage disease patients, specially Niemann-Pick disease type C (Kaya …

N-acetyl-L-leucine improves symptoms and functioning in GM2 Gangliosidosis (Tay-Sachs & Sandhoff)

K Martakis, J Claassen, J Gascon-Bayarri… - medRxiv, 2021 - medrxiv.org
lysosomal storage disorders like Niemann-Pick disease type … have shown that N-acetyl-leucine
significantly reduced ataxia … , slowed disease progression, and prolonged survival. These …