CLINICAL STUDIES

Title
Author(s)
Summary
Strupp, M., Bayer, O., Feil, K., Straube, A.

A paper demonstrating the significant benefit of N-Acetyl-DL-Leucine for the prophylactic treatment of migraine, as well as migraine with aura. Journal of Neurology. 

Cortina-Borja,M., te Vruchte,D., Mengel,E., Amraoui,Y., Imrie, J. ,Jones,S., Dali,C., Fineran,P., Kirkegaard,T., Runz,H., Lachmann,R., Bremova-Ertl,T., Strupp,M., Platt,F.

Demonstrates the annual severity increment score (ASIS) has prognostic value in assessing the progression of Niemann-Pick type C, and that long-term treatment with N-Acetyl-DL-leucine is associated with a reduction in ASIS scores. Orphanet Journal of Rare Diseases 13, 1-16 (2018).

Platt, F., Strupp, M.

A case study demonstrating that, in addition to being of benefit in patients with cerebellar ataxia and Niemann-Pick disease type C, acetyl-DL-leucine can increase mobility and quality of life in a healthy elderly subject.Journal of Neurology 263, 1239-40 (2016).

Schniepp, R., Strupp, M. Wuehr, M. Jahn, K. Dieterich, M. Brandt, T. Feil, K.

A paper exhibiting the significant benefit of Acetyl-DL-Leucine in the treatment of Gait variability in patients with cerebellar ataxia. Cerebellum & Ataxias 3:8 (2016).

Strupp M, Teufel J, Habs M, Feuerecker R, Muth C, van de Warrenburg BP, Klopstock T, Feil K.

A paper exhibiting the significant benefit of Acetyl-DL-Leucine in the treatment of patients with cerebellar ataxia.Journal of Neurology 260, 2556-2561 (2013).

Bremova T, Malinová V, Amraoui Y, Mengel E, Reinke J, Kolníková M, Strupp M.

Demonstrates that Acetyl-DL-Leucine treatment is associated with improvements in ataxia and quality of life in patients with Niemann-Pick Disease Type C.Neurology 85, 1368-1375 (2015).

Ferber-Viart C, Dubreuil C, Vidal PP. Audiol Neurootol.

2009;14(1):17-25. doi: 10.1159/000148206. Epub 2008 Jul 29. PMID: 18663295
Acetyl-DL-leucine improves gait variability in patients with cerebellar ataxia—a case series Schniepp et al. Cerebellum & Ataxias (2016) 3:8 DOI 10.1186/s40673-016-0046-2
Gait ataxia in humans: vestibular and cerebellar control of dynamic stability J Neurol (2017) 264 (Suppl 1):S87–S92 DOI 10.1007/s00415-017-8482-3

NON-CLINICAL STUDIES

Title
Author(s)
Summary
te Vrutche, D., Galione, A., Strupp, M., Mann, M.

N-Acetyl-DL-Leucine and N-Acetyl-L-Leucine reduced the relative lysosomal volume in NPC1-/- Chinese Hamster Ovary cells in a dose-dependent manner. N-Acetyl-L-Leucine was most effective at reducing relative lysosomal volumes in fibroblasts derived from NPC patients with severe disease (***p <0.001), followed by N-Acetyl-DL-Leucine (**p <0.01). Treatment with N-Acetyl-D-Leucine did not achieve statistical significance.

Sarkar, C., Hegdekar, N., Lipinski, M.M

bioRxiv, 2019. https://doi.org/10.1101/759894

Treatment with N-acetyl-L-leucine is expected to be beneficial in restricting neuronal death and hence improving neurological function after Traumatic Brain Injury (TBI) and is a promising, novel, neuroprotective drug candidate for the treatment of TBI.

Churchill C., Strupp M., Galione A., Platt F.

bioRxiv, 2019. https://doi.org/10.1101/738286

Kalla, R., Teufel, J., Feil, K., Strupp, M.

Journal of Neurology 263 (2016).

Feil K, Bremova T, Muth C, Schniepp R, Teufel J, Strupp M.

Cerebellum.15(1):38-42. (2016).

Günther L, Beck R, Xiong G, Potschka H, Jahn K, Bartenstein P, Brandt T, Dutia M, Dieterich M, Strupp M, la Fougère C, Zwergal A.

PLoS One. 2015 Mar 24;10(3):e0120891. doi: 10.1371/journal.pone.0120891. eCollection 2015. PMID: 25803613