Clinical Studies

Acetyl-Leucine Improves Cerebellar Dysarthria in MSA-C

Lehner, L & Strupp, M.
Pub Med

“How to save a marriage”: treatment of REM sleep behavior disorder (RBD) with acetyl-leucine in a patient with Parkinson’s disease

Oertel, W., Henrich, M., Bergquist, F., Janzen A. Geibl, F.F., Michael, S.
Pub Med

Therapeutic potential of acetyl-DL-leucine and its L-enantiomer in posterior fossa syndrome: Mechanistic insights

Vlček, P., Horáček, J., Grünerová-Lippertová, M., Brunovský, M.
Pub Med

Disease-Modifying, Neuroprotective Effect of N-Acetyl-l-Leucine in Adult and Pediatric Patients With Niemann-Pick Disease Type C

Patterson, M., Ramaswami, U., Donald, A., Foltan, T., Gautschi, M., Gissen, P., Hahn, A., Jones, S., Kay, R., Kolnikova, M., Park, J., Reichmannova, S., Walterfang, M., Wibawa, P., Rohrbach, M., Martakis, K., Bremova-Ertl, T.
Pub Med

Tourette syndrome: effects of acetyl-leucine in two individuals

Hofer, E.M., Mosil, R., Strupp, L., Falkai, P., Strupp, M., Schindlbeck, K.A.
Pub Med

Trends in Pharmacological Sciences Drug of the Month: Levacetylleucine (N-acetyl-L-leucine) for Niemann-Pick disease type C

van Gool, R., Al-Hertani, W., Bodamer, O., Upadhyay, J.
Pub Med

N-Acetyl-leucine in progressive CACNA1A ataxia: A case series

Martakis, K., Giorgi, M., Spanou, M., Neubauer, B.A., Dinopoulosc, D., Hahn, A.
Pub Med

Acetyl-DL-leucine in two individuals with REM sleep behavior disorder improves symptoms, reverses loss of striatal dopamine-transporter binding and stabilizes pathological metabolic brain pattern-case reports

OERTEL, W.H., JANZEN, A., HENRICH, M.T., GEIBL, F.F., SITTIG, E., MELES, S.K., CARLI, G., LEENDERS, K., BOOJI, J., SURMEIER, J., TIMMERMANN, L., STRUPP, M.
Pub Med

N-ACETYL-L-LEUCINE AND NEURODEGENERATIVE DISEASE

TIFFT, C.J
Pub Med

IB1001 Pivotal Trial Published in NEJM

TATIANA BREMOVA-ERTL, M.D., PH.D., UMA RAMASWAMI, M.D., MARION BRANDS, M.D., TOMAS FOLTAN, M.D., MATTHIAS GAUTSCHI, M.D., PH.D., PAUL GISSEN, M.D., FRANCESCA GOWING, M.D., ANDREAS HAHN, M.D., SIMON JONES, M.D., RICHARD KAY, PH.D., MIRIAM KOLNIKOVA, M.D., PH.D., LAILA ARASH-KAPS, M.D., THORSTEN MARQUARDT, M.D., EUGEN MENGEL, M.D., JULIEN H. PARK, M.D., STELLA REICHMANNOVÁ, M.D., PH.D., SUSANNE A. SCHNEIDER, M.D., SIYAMINI SIVANANTHAN, M.D., MARK WALTERFANG, M.D., PH.D., D.MED.SCI., PIERRE WIBAWA, M.D., MICHAEL STRUPP, M.D., AND KYRIAKOS MARTAKIS, M.D.
Pub Med

N-ACETYL-L-LEUCINE FOR NIEMANN-PICK TYPE C: A MULTINATIONAL DOUBLE-BLIND RANDOMIZED PLACEBO-CONTROLLED CROSSOVER STUDY

FIELDS, T., BREMOVA-ERTL, T., BILLINGTON, I., CHURCHILL, G.C., EVANS, W., FIELDS, C., GALIONE, A., KAY, R., MATHIESON, T., MARTAKIS, K., PATTERSON, M., PLATT, F., FACTOR, M., STRUPP, M.
Pub Med

ACETYL-DL-LEUCINE IN COMBINATION WITH MEMANTINE IMPROVES ACQUIRED PENDULAR NYSTAGMUS CAUSED BY MULTIPLE SCLEROSIS: A CASE REPORT

KREMMYDA, O., FEIL, K., BARDINS,S., STRUPP, M.
Pub Med

THE EFFECT OF N-ACETYL-DL-LEUCINE ON NEUROLOGICAL SYMPTOMS IN A PATIENT WITH ATAXIA-TELANGIECTASIA: A CASE STUDY

SABERI-KARIMIAN, M., BEYRAGHI-TOUSI, M., MIRZADEH, M. GUMPRICHT, E., SAHEBKAR, A.
Pub Med

EFFECTS OF ACETYL-DL-LEUCINE ON ATAXIA AND DOWNBEAT-NYSTAGMUS IN SIX PATIENTS WITH ATAXIA TELANGIECTASIA

BRUEGGEMANN, A., BICVIC, A., GOELDLIN, M., KALLA, R., KERKENI, H., MANTOKOUDIS, G., ABEGG, M., KOLNIKOVA, M., MOHAUPT, M., BREMOVA-ERTL, T.
Pub Med

EFFICACY AND SAFETY OF N-ACETYL-L-LEUCINE IN CHILDREN AND ADULTS WITH GM2 GANGLIOSIDOSES

MARTAKIS, K., CLAASSEN, J., GASCON BAYARRI, J., GOLDSCHAGG,N., HAHN, A., HASSAN, A., HENNIG, A., JONES, S. , LAU, H., PERLMAN, S., SHARMA, R., SCHNEIDER, S. A., BREMOVA-ERTL. T
Pub Med

EFFICACY AND SAFETY OF N-ACETYL-L-LEUCINE IN NIEMANN-PICK DISEASE TYPE

BREMOVA, T., CLAASSEN, J., FOLTAN, T., GASCON BAYARRI, J., GISSEN, P., HAHN, A., HASSAN, A., HENNIG, A., JONES, S. , KOLNIKOVA, M., MARTAKIS, K., RAMASWAMI, U, SHARMA, R., SCHNEIDER, S. A.
Pub Med

SANDHOFF DISEASE: IMPROVEMENT OF GAIT BY ACETYL-DL-LEUCINE: A CASE REPORT

BREMOVA-ERTL, T., PLATT, F., STRUPP, M.
Pub Med

Non-clinical Studies

N-acetyl-l-leucine lowers pS129-synuclein and improves synaptic function in models of Parkinson’s disease

Song, P., Franchini, R., Chen, C. Duong, B., Wang, YZ., Savas, J. Parisiadou. L, Krainc, D.
Pub Med

Stereospecific rapid activation of Transcription Factor EB (TFEB) by Levacetylleucine (NALL)

Davis, L.C, Brain,R., Churchill, G., Factor, M., Fields, T., Platt, F., Patterson, M., Strupp, P., Galione, A.
Pub Med

N-Acetyl-l-Leucine (NALL) rescues inter-organelle communication in Niemann-Pick disease type-C patient cells

Kiraly, S., Martello, A., Platt, F., Eden, E.
Pub Med

Myeloid cell–specific loss of NPC1 in mice recapitulates microgliosis and neurodegeneration in patients with Niemann-Pick type C disease

Lina Dinkel, Selina Hummel, Valerio Zenatti, Mariagiovanna Malara, Yannik Tillmann, Alessio Colombo, Laura Sebastian Monasor, Jung H. Suh, Todd Logan, Stefan Roth, Lars Paeger, Patricia Hoffelner, Oliver Bludau, Andree Schmidt, Stephan A. Müller, Martina Schiffler, Brigitte Nuscher, Jasenka Rudan Njavro, Matthias Prestel, Laura M. Bartos, Karin Wind-Mark, Luna Slemann, Leonie Hoermann, Sebastian T. Kunte, Johannes Gnörich, Simon Lindner, Mikael Simons, Jochen Herms, Dominik Paquet, Stefan F. Lichtenthaler, Peter Bartenstein, Nicolai Franzmeier, Arthur Liesz, Antje Grosche, Tatiana Bremova-Ertl, Claudia Catarino, Skadi Beblo, Caroline Bergner Susanne A. Schneider, Michael Strupp, Gilbert Di Paolo, Matthias Brendel, Sabina Tahirovic
Pub Med

ORMDL MISLOCALIZATION BY IMPAIRED AUTOPHAGY IN NIEMANN-PICK TYPE C DISEASE LEADS TO INCREASED DE NOVO SPHINGOLIPID BIOSYNTHESIS

BROWN, R., MAHAWAR, U., WATTENBERG, B., SPIEGEL, S.
Pub Med

N-ACETYL-L-LEUCINE PROTECTS MPTP-TREATED PARKINSON’S DISEASE MOUSE MODELS BY SUPPRESSING DESULFOBACTEROTA VIA THE GUT-BRAIN AXIS

ZHIFENG XUA, CHANGLIN LIANA, LIXIN PANA, WENJIE LAIA,E, FEN ZHANGA, LINGMEI PENGA, SIJIE ZHOU B, GUANGHUA ZHAO C, XUEZHU YANG D, GUOHUA ZHANG A, E, ZEFENG TAN A, YUKAI WANGA
Pub Med

EFFICIENT NEUROPROTECTIVE RESCUE OF SACSIN-RELATED DISEASE PHENOTYPES IN ZEBRAFISH

NAEF, V., MARCHESE, M., OGI, A., FICHI, G., GALATOLO, D., LICITRA, R., DOCCINI, S., VERRI, T., ARGENTON, F., MORANI, F., SANTORELLI, F.
Pub Med

ACETYLATION TURNS LEUCINE INTO A DRUG BY MEMBRANE TRANSPORTER SWITCHING

CHURCHILL, C., STRUPP, M., FACTOR, C., BREMOVA-ERTL, T., FACTOR, M., PATTERSON, M., PLATT, F.M., GALIONE, A.
Pub Med

ACETYLATION OF L-LEUCINE SWITCHES ITS CARRIER FROM THE L-AMINO ACID TRANSPORTER (LAT) TO ORGANIC ANION TRANSPORTERS (OAT)

CHURCHILL, C., STRUPP, M., BREMOVA-ERTL, T., FACTOR, M., PATTERSON, M., PLATT, F., GALIONE, A.
Pub Med

ACETYL-LEUCINE SLOWS DISEASE PROGRESSION IN LYSOSOMAL STORAGE DISORDERS

KAYA, E., SMITH, D., SMITH, C., MORRIS, L., BREMOVA-ERTL, T., CORTINA-BORJA, M., FINERAN, P., MORTEN, K., POULTON, J., BOLAND, B., SPENCER, J., STRUPP, M., PLATT, F.M.
Pub Med

BENEFICIAL EFFECTS OF ACETYL-DL-LEUCINE (ADLL) IN A MOUSE MODEL OF SANDHOFF DISEASE

KAYA, E., SMITH, D.A, SMITH, C., BOLAND, B. STRUPP, M., PLATT, F.M.
Pub Med

EFFECTS OF N-ACETYL-LEUCINE AND ITS ENANTIOMERS IN NIEMANN-PICK DISEASE TYPE C CELLS

TE VRUTCHE, D., GALIONE, A., STRUPP, M., MANN, M.
Pub Med

N‐ACETYL‐L‐LEUCINE IMPROVES FUNCTIONAL RECOVERY AND ATTENUATES CORTICAL CELL DEATH AND NEUROINFLAMMATION AFTER TRAUMATIC BRAIN INJURY IN MICE

HEGDEKAR, N., LIPINSKI, M.M, SARKAR, C.
Pub Med

UNEXPECTED DIFFERENCES IN THE PHARMACOKINETICS OF N-ACETYL DL-LEUCINE ENANTIOMERS AFTER ORAL DOSING AND THEIR CLINICAL RELEVANCE

CHURCHILL C., STRUPP M., GALIONE A., PLATT F.
Pub Med

UPDATE ON THE PHARMACOTHERAPY OF CEREBELLAR AND CENTRAL VESTIBULAR DISORDERS.

KALLA, R., TEUFEL, J., FEIL, K., STRUPP, M.
Pub Med
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