Clinical Studies

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

THE EFECTS OF N‑ACETYL‑L‑LEUCINE ON THE IMPROVEMENT OF SYMPTOMS IN A PATIENT WITH MULTIPLE SULFATASE DEFCIENCY

SABERI-KARIMIAN, M., HOURA, M. JAMIALAHMADI, T., SARVGHADI, P., NIKBAF, M., AKHLAGHI, S., SAHEBKAR, A.
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

Non-clinical Studies

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
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