ambroxol has been researched along with Gaucher Disease in 27 studies
Ambroxol: A metabolite of BROMHEXINE that stimulates mucociliary action and clears the air passages in the respiratory tract. It is usually administered as the hydrochloride.
Gaucher Disease: An autosomal recessive disorder caused by a deficiency of acid beta-glucosidase (GLUCOSYLCERAMIDASE) leading to intralysosomal accumulation of glycosylceramide mainly in cells of the MONONUCLEAR PHAGOCYTE SYSTEM. The characteristic Gaucher cells, glycosphingolipid-filled HISTIOCYTES, displace normal cells in BONE MARROW and visceral organs causing skeletal deterioration, hepatosplenomegaly, and organ dysfunction. There are several subtypes based on the presence and severity of neurological involvement.
| Excerpt | Relevance | Reference |
|---|---|---|
| "The purpose of this pilot was to assess the tolerability and efficacy of ambroxol as a pharmacological chaperone in patients with symptomatic, type 1 Gaucher disease who present with measurable disease parameters but are not receiving enzyme replacement therapy (ERT) in order to provide proof of concept and/or ascertain the suitability of ambroxol for a larger clinical trial." | 9.17 | Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease. ( Altarescu, G; Elstein, D; Zimran, A, 2013) |
| "To assess hematologic and visceral outcomes, biomarker changes, and safety of ambroxol therapy for patients with Gaucher disease (GD) without disease-specific treatment." | 8.31 | Use of Ambroxol as Therapy for Gaucher Disease. ( Gao, X; Li, J; Maegawa, GHB; Wang, D; Wang, Y; Zhan, X; Zhang, H, 2023) |
| " Recently, several studies have shown a promising outcome of ambroxol chaperone therapy for the treatment of GD, but its therapeutic role in GD1-associated liver cirrhosis and portal hypertension was not verified." | 8.12 | Ambroxol Chaperone Therapy for Gaucher Disease Type I-Associated Liver Cirrhosis and Portal Hypertension: A Case Report. ( Cui, SY; Gao, RP; Zhang, P; Zhang, W; Zheng, MF, 2022) |
| "Ambroxol (ABX) has been suggested as an augmentative pharmacological agent for neuronopathic Gaucher disease (nGD)." | 7.96 | Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy. ( Bae, JS; Cozma, C; Heo, SH; Jin, HK; Kim, HW; Kim, T; Kim, YM; Ko, TS; Lee, BH; Lim, HS; Lim, HT; Oh, A; Osborn, MJ; Rolfs, A; Seo, GH; Tolar, J; Yoo, HW; Yoon, HM; Yum, MS; Zimran, A, 2020) |
| "Both patients with non-neuronopathic Gaucher disease (GD) and heterozygous GBA mutation carrier are at increased risk for Parkinson disease (PD)." | 6.58 | Combined beta-glucosylceramide and ambroxol hydrochloride in patients with Gaucher related Parkinson disease: From clinical observations to drug development. ( Arkadir, D; Dinur, T; Ilan, Y; Ishay, Y; Szer, J; Zimran, A, 2018) |
| "Ambroxol hydrochloride is an oral mucolytic drug available over-the-counter for many years as cough medicine." | 5.62 | Upgrading the evidence for the use of ambroxol in Gaucher disease and GBA related Parkinson: Investigator initiated registry based on real life data. ( Al-Hertani, W; Becker-Cohen, M; Castillo-Garcia, D; Ceron-Rodriguez, M; Chan, A; Dinur, T; Fiumara, A; Istaiti, M; Kiec-Wilk, B; Lee, BH; Ramaswami, U; Revel-Vilk, S; Rodic, P; Rubio, B; Tincheva, RS; Yang, CF; Zimran, A, 2021) |
| "Ambroxol treatment significantly reduced HexSph concentration in GD (by 2." | 5.62 | Ambroxol increases glucocerebrosidase (GCase) activity and restores GCase translocation in primary patient-derived macrophages in Gaucher disease and Parkinsonism. ( Baydakova, GV; Bezrukikh, VA; Bogdanova, DA; Bolshakova, OI; Cheblokov, AA; Emelyanov, AK; Kopytova, AE; Miliukhina, IV; Nikolaev, MA; Pchelina, SN; Rychkov, GN; Salogub, GN; Sarantseva, SV; Senkevich, KA; Usenko, TC; Zakharova, EY, 2021) |
| "Ambroxol has been suggested as an enzyme enhancement agent." | 5.62 | Early initiation of ambroxol treatment diminishes neurological manifestations of type 3 Gaucher disease: A long-term outcome of two siblings. ( Barić, I; Bogdanić, A; Bošnjak-Nađ, K; Fumić, K; Mustać, G; Ohno, K; Ozretić, D; Ramadža, DP; Škaričić, A; Zekušić, M; Žigman, T, 2021) |
| "Treatment with ambroxol hydrochloride increased glucosylceramidase activity in fibroblasts from healthy controls, Gaucher disease and heterozygous glucocerebrosidase mutation carriers with and without Parkinson's disease." | 5.40 | Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells. ( Abramov, AY; Chau, KY; Cooper, JM; Foltynie, T; Gegg, M; Hughes, D; Magalhaes, J; McNeill, A; Mehta, A; Schapira, AH; Shen, C, 2014) |
| " The literature review of the usage of ambroxol acting as a pharmacological chaperone for beta-glucocerebrosidase in Gaucher disease and Parkinson’s disease associated with GBA variants has been reviewed." | 5.22 | Pharmacological chaperone therapy for the treatment of inborn errors of metabolism ( Jezela-Stanek, A; Lipiński, P; Tylki-Szymańska, A, 2022) |
| "The purpose of this pilot was to assess the tolerability and efficacy of ambroxol as a pharmacological chaperone in patients with symptomatic, type 1 Gaucher disease who present with measurable disease parameters but are not receiving enzyme replacement therapy (ERT) in order to provide proof of concept and/or ascertain the suitability of ambroxol for a larger clinical trial." | 5.17 | Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease. ( Altarescu, G; Elstein, D; Zimran, A, 2013) |
| "To assess hematologic and visceral outcomes, biomarker changes, and safety of ambroxol therapy for patients with Gaucher disease (GD) without disease-specific treatment." | 4.31 | Use of Ambroxol as Therapy for Gaucher Disease. ( Gao, X; Li, J; Maegawa, GHB; Wang, D; Wang, Y; Zhan, X; Zhang, H, 2023) |
| " Recently, several studies have shown a promising outcome of ambroxol chaperone therapy for the treatment of GD, but its therapeutic role in GD1-associated liver cirrhosis and portal hypertension was not verified." | 4.12 | Ambroxol Chaperone Therapy for Gaucher Disease Type I-Associated Liver Cirrhosis and Portal Hypertension: A Case Report. ( Cui, SY; Gao, RP; Zhang, P; Zhang, W; Zheng, MF, 2022) |
| "Ambroxol (ABX) has been suggested as an augmentative pharmacological agent for neuronopathic Gaucher disease (nGD)." | 3.96 | Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy. ( Bae, JS; Cozma, C; Heo, SH; Jin, HK; Kim, HW; Kim, T; Kim, YM; Ko, TS; Lee, BH; Lim, HS; Lim, HT; Oh, A; Osborn, MJ; Rolfs, A; Seo, GH; Tolar, J; Yoo, HW; Yoon, HM; Yum, MS; Zimran, A, 2020) |
| "Our work supports the proposition that ambroxol should be further investigated as a potential novel disease-modifying therapy for treatment of Parkinson disease and neuronopathic Gaucher disease to increase glucocerebrosidase activity and decrease α-synuclein and phosphorylated α-synuclein protein levels." | 3.83 | Ambroxol effects in glucocerebrosidase and α-synuclein transgenic mice. ( Bezard, E; Daly, L; Migdalska-Richards, A; Schapira, AH, 2016) |
| "Both patients with non-neuronopathic Gaucher disease (GD) and heterozygous GBA mutation carrier are at increased risk for Parkinson disease (PD)." | 2.58 | Combined beta-glucosylceramide and ambroxol hydrochloride in patients with Gaucher related Parkinson disease: From clinical observations to drug development. ( Arkadir, D; Dinur, T; Ilan, Y; Ishay, Y; Szer, J; Zimran, A, 2018) |
| "Ambroxol (ABX) is a mucolytic agent used for the treatment of respiratory diseases." | 1.72 | Mechanistic Insight into the Mode of Action of Acid β-Glucosidase Enhancer Ambroxol. ( Cubellis, MV; Hermann, A; Hules, L; Lukas, J; Monticelli, M; Pantoom, S; Petrosyan, A; Pospech, J; Schöll, C, 2022) |
| "Ambroxol hydrochloride is an oral mucolytic drug available over-the-counter for many years as cough medicine." | 1.62 | Upgrading the evidence for the use of ambroxol in Gaucher disease and GBA related Parkinson: Investigator initiated registry based on real life data. ( Al-Hertani, W; Becker-Cohen, M; Castillo-Garcia, D; Ceron-Rodriguez, M; Chan, A; Dinur, T; Fiumara, A; Istaiti, M; Kiec-Wilk, B; Lee, BH; Ramaswami, U; Revel-Vilk, S; Rodic, P; Rubio, B; Tincheva, RS; Yang, CF; Zimran, A, 2021) |
| "Ambroxol treatment significantly reduced HexSph concentration in GD (by 2." | 1.62 | Ambroxol increases glucocerebrosidase (GCase) activity and restores GCase translocation in primary patient-derived macrophages in Gaucher disease and Parkinsonism. ( Baydakova, GV; Bezrukikh, VA; Bogdanova, DA; Bolshakova, OI; Cheblokov, AA; Emelyanov, AK; Kopytova, AE; Miliukhina, IV; Nikolaev, MA; Pchelina, SN; Rychkov, GN; Salogub, GN; Sarantseva, SV; Senkevich, KA; Usenko, TC; Zakharova, EY, 2021) |
| "Ambroxol has been suggested as an enzyme enhancement agent." | 1.62 | Early initiation of ambroxol treatment diminishes neurological manifestations of type 3 Gaucher disease: A long-term outcome of two siblings. ( Barić, I; Bogdanić, A; Bošnjak-Nađ, K; Fumić, K; Mustać, G; Ohno, K; Ozretić, D; Ramadža, DP; Škaričić, A; Zekušić, M; Žigman, T, 2021) |
| "Treatment with ambroxol hydrochloride increased glucosylceramidase activity in fibroblasts from healthy controls, Gaucher disease and heterozygous glucocerebrosidase mutation carriers with and without Parkinson's disease." | 1.40 | Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells. ( Abramov, AY; Chau, KY; Cooper, JM; Foltynie, T; Gegg, M; Hughes, D; Magalhaes, J; McNeill, A; Mehta, A; Schapira, AH; Shen, C, 2014) |
| "Gaucher disease is an autosomal recessively inherited storage disorder caused by deficiency of the lysosomal hydrolase, acid β-glucosidase." | 1.39 | Transgenic mice expressing human glucocerebrosidase variants: utility for the study of Gaucher disease. ( Clarke, LA; Fuller, M; Hein, L; Hemmelgarn, H; Melrose, HL; Sanders, A, 2013) |
| " No serious adverse effect was observed during this experiment." | 1.39 | The chaperone activity and toxicity of ambroxol on Gaucher cells and normal mice. ( Higaki, K; Li, L; Luan, Z; Nanba, E; Ohno, K; Suzuki, Y, 2013) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 1 (3.70) | 29.6817 |
| 2010's | 14 (51.85) | 24.3611 |
| 2020's | 12 (44.44) | 2.80 |
| Authors | Studies |
|---|---|
| Castilla, J | 1 |
| Rísquez, R | 1 |
| Cruz, D | 1 |
| Higaki, K | 3 |
| Nanba, E | 3 |
| Ohno, K | 3 |
| Suzuki, Y | 2 |
| Díaz, Y | 1 |
| Ortiz Mellet, C | 2 |
| García Fernández, JM | 2 |
| Castillón, S | 1 |
| García-Moreno, MI | 1 |
| de la Mata, M | 1 |
| Sánchez-Fernández, EM | 1 |
| Benito, JM | 1 |
| Díaz-Quintana, A | 1 |
| Fustero, S | 1 |
| Sánchez-Alcázar, JA | 1 |
| Zhang, P | 1 |
| Zheng, MF | 1 |
| Cui, SY | 1 |
| Zhang, W | 1 |
| Gao, RP | 1 |
| Pantoom, S | 1 |
| Hules, L | 1 |
| Schöll, C | 1 |
| Petrosyan, A | 1 |
| Monticelli, M | 1 |
| Pospech, J | 1 |
| Cubellis, MV | 1 |
| Hermann, A | 1 |
| Lukas, J | 1 |
| Lipiński, P | 1 |
| Jezela-Stanek, A | 1 |
| Tylki-Szymańska, A | 1 |
| Zhan, X | 1 |
| Zhang, H | 2 |
| Maegawa, GHB | 2 |
| Wang, Y | 1 |
| Gao, X | 1 |
| Wang, D | 1 |
| Li, J | 1 |
| Weinreb, NJ | 1 |
| Goker-Alpan, O | 2 |
| Kim, YM | 1 |
| Yum, MS | 1 |
| Heo, SH | 1 |
| Kim, T | 1 |
| Jin, HK | 1 |
| Bae, JS | 1 |
| Seo, GH | 1 |
| Oh, A | 1 |
| Yoon, HM | 1 |
| Lim, HT | 1 |
| Kim, HW | 1 |
| Ko, TS | 1 |
| Lim, HS | 1 |
| Osborn, MJ | 1 |
| Tolar, J | 1 |
| Cozma, C | 1 |
| Rolfs, A | 1 |
| Zimran, A | 4 |
| Lee, BH | 2 |
| Yoo, HW | 1 |
| Jiang, W | 1 |
| Yi, M | 1 |
| Chu, SY | 1 |
| Chien, CC | 1 |
| Hwu, WL | 1 |
| Wang, PJ | 1 |
| Chien, YH | 1 |
| Pawlinski, L | 2 |
| Krawczyk, M | 1 |
| Fiema, M | 1 |
| Tobor, E | 1 |
| Kiec-Wilk, B | 3 |
| Istaiti, M | 1 |
| Revel-Vilk, S | 1 |
| Becker-Cohen, M | 1 |
| Dinur, T | 2 |
| Ramaswami, U | 1 |
| Castillo-Garcia, D | 1 |
| Ceron-Rodriguez, M | 1 |
| Chan, A | 1 |
| Rodic, P | 1 |
| Tincheva, RS | 1 |
| Al-Hertani, W | 1 |
| Yang, CF | 1 |
| Fiumara, A | 1 |
| Rubio, B | 1 |
| Kopytova, AE | 1 |
| Rychkov, GN | 1 |
| Nikolaev, MA | 1 |
| Baydakova, GV | 1 |
| Cheblokov, AA | 1 |
| Senkevich, KA | 1 |
| Bogdanova, DA | 1 |
| Bolshakova, OI | 1 |
| Miliukhina, IV | 1 |
| Bezrukikh, VA | 1 |
| Salogub, GN | 1 |
| Sarantseva, SV | 1 |
| Usenko, TC | 1 |
| Zakharova, EY | 1 |
| Emelyanov, AK | 1 |
| Pchelina, SN | 1 |
| Ramadža, DP | 1 |
| Zekušić, M | 1 |
| Žigman, T | 1 |
| Škaričić, A | 1 |
| Bogdanić, A | 1 |
| Mustać, G | 1 |
| Bošnjak-Nađ, K | 1 |
| Ozretić, D | 1 |
| Fumić, K | 1 |
| Barić, I | 1 |
| Thirumal Kumar, D | 1 |
| Iyer, S | 1 |
| Christy, JP | 1 |
| Siva, R | 1 |
| Tayubi, IA | 1 |
| George Priya Doss, C | 1 |
| Zayed, H | 1 |
| Sanders, A | 1 |
| Hemmelgarn, H | 1 |
| Melrose, HL | 1 |
| Hein, L | 1 |
| Fuller, M | 2 |
| Clarke, LA | 1 |
| McNeill, A | 1 |
| Magalhaes, J | 1 |
| Shen, C | 1 |
| Chau, KY | 1 |
| Hughes, D | 1 |
| Mehta, A | 1 |
| Foltynie, T | 1 |
| Cooper, JM | 1 |
| Abramov, AY | 1 |
| Gegg, M | 1 |
| Schapira, AH | 2 |
| Albin, RL | 1 |
| Dauer, WT | 1 |
| Maor, G | 2 |
| Cabasso, O | 1 |
| Krivoruk, O | 1 |
| Rodriguez, J | 1 |
| Steller, H | 1 |
| Segal, D | 1 |
| Horowitz, M | 2 |
| Migdalska-Richards, A | 1 |
| Daly, L | 1 |
| Bezard, E | 1 |
| Ishay, Y | 1 |
| Szer, J | 1 |
| Ilan, Y | 1 |
| Arkadir, D | 1 |
| Malecki, MT | 1 |
| Maegawa, GH | 1 |
| Tropak, MB | 1 |
| Buttner, JD | 1 |
| Rigat, BA | 1 |
| Pandit, D | 1 |
| Tang, L | 1 |
| Kornhaber, GJ | 1 |
| Hamuro, Y | 1 |
| Clarke, JT | 1 |
| Mahuran, DJ | 1 |
| Luan, Z | 1 |
| Li, L | 1 |
| Altarescu, G | 1 |
| Elstein, D | 1 |
| Bendikov-Bar, I | 1 |
| Filocamo, M | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| Effect of Ambroxol on the Inflammatory Markers and Clinical Outcome of Patients With Diabetic Peripheral Neuropathy[NCT05558878] | 80 participants (Anticipated) | Interventional | 2022-10-01 | Not yet recruiting | |||
| Ambroxol as a Disease-modifying Treatment to Reduce the Risk of Cognitive Impairment in GBA-associated Parkinson's Disease. A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 2 Trial[NCT05287503] | Phase 2 | 65 participants (Actual) | Interventional | 2022-02-15 | Active, not recruiting | ||
| Genotypic Influences on Network Progression in Parkinson's Disease[NCT04228172] | 32 participants (Anticipated) | Observational | 2020-02-24 | Recruiting | |||
| Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy[NCT03950050] | Phase 2 | 40 participants (Actual) | Interventional | 2019-03-01 | Completed | ||
| Ambroxol as a Novel Disease Modifying Treatment for Parkinson's Disease Dementia[NCT02914366] | Phase 2 | 55 participants (Actual) | Interventional | 2015-11-30 | Active, not recruiting | ||
| Ambroxol as a Novel Disease Modifying Treatment for Lewy Body Dementia[NCT04405596] | Phase 1/Phase 2 | 15 participants (Anticipated) | Interventional | 2025-01-31 | Not yet recruiting | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
3 reviews available for ambroxol and Gaucher Disease
| Article | Year |
|---|---|
Pharmacological chaperone therapy for the treatment of inborn errors of metabolism
Topics: Ambroxol; Gaucher Disease; Humans; Mutation; Parkinson Disease | 2022 |
A comparative computational approach toward pharmacological chaperones (NN-DNJ and ambroxol) on N370S and L444P mutations causing Gaucher's disease.
Topics: 1-Deoxynojirimycin; Ambroxol; Gaucher Disease; Glucosylceramidase; Humans; Models, Molecular; Molecu | 2019 |
Combined beta-glucosylceramide and ambroxol hydrochloride in patients with Gaucher related Parkinson disease: From clinical observations to drug development.
Topics: Ambroxol; Animals; Anti-Inflammatory Agents; Drug Discovery; Expectorants; Gaucher Disease; Glucosyl | 2018 |
1 trial available for ambroxol and Gaucher Disease
| Article | Year |
|---|---|
Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease.
Topics: Adult; Ambroxol; Drug Hypersensitivity; Enzyme Stability; Female; Gaucher Disease; Glucosylceramidas | 2013 |
Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease.
Topics: Adult; Ambroxol; Drug Hypersensitivity; Enzyme Stability; Female; Gaucher Disease; Glucosylceramidas | 2013 |
Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease.
Topics: Adult; Ambroxol; Drug Hypersensitivity; Enzyme Stability; Female; Gaucher Disease; Glucosylceramidas | 2013 |
Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease.
Topics: Adult; Ambroxol; Drug Hypersensitivity; Enzyme Stability; Female; Gaucher Disease; Glucosylceramidas | 2013 |
23 other studies available for ambroxol and Gaucher Disease
| Article | Year |
|---|---|
Conformationally-locked N-glycosides with selective β-glucosidase inhibitory activity: identification of a new non-iminosugar-type pharmacological chaperone for Gaucher disease.
Topics: Cells, Cultured; Fibroblasts; Gaucher Disease; Glucosylceramidase; Glycosides; Humans; Molecular Con | 2012 |
Fluorinated Chaperone-β-Cyclodextrin Formulations for β-Glucocerebrosidase Activity Enhancement in Neuronopathic Gaucher Disease.
Topics: beta-Cyclodextrins; Cells, Cultured; Fluorine; Gaucher Disease; Glucosylceramidase; Humans; Molecula | 2017 |
Ambroxol Chaperone Therapy for Gaucher Disease Type I-Associated Liver Cirrhosis and Portal Hypertension: A Case Report.
Topics: Ambroxol; Gaucher Disease; Glucosylceramidase; Humans; Hypertension, Portal; Liver Cirrhosis; Male | 2022 |
Mechanistic Insight into the Mode of Action of Acid β-Glucosidase Enhancer Ambroxol.
Topics: Ambroxol; beta-Glucosidase; Gaucher Disease; Glucosylceramidase; Humans; Molecular Chaperones | 2022 |
Use of Ambroxol as Therapy for Gaucher Disease.
Topics: Adolescent; Ambroxol; Biomarkers; Child; Child, Preschool; China; Gaucher Disease; Hemoglobins; Huma | 2023 |
Ambroxol as Therapy for Gaucher Disease-Ambitious but Ambivalent.
Topics: Ambroxol; Gaucher Disease; Glucosylceramidase; Humans; Mutation | 2023 |
Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy.
Topics: Adolescent; Ambroxol; Biomarkers; Child; Dose-Response Relationship, Drug; Enzyme Replacement Therap | 2020 |
Ambroxol improves skeletal and hematological manifestations on a child with Gaucher disease.
Topics: Ambroxol; Child; Enzyme Replacement Therapy; Gaucher Disease; Glucosylceramidase; Humans; Lysosomal | 2020 |
Early initiation of high-dose oral ambroxol in combination with enzyme replacement therapy in a neuropathic Gaucher infant.
Topics: Ambroxol; Drug Therapy, Combination; Enzyme Replacement Therapy; Gaucher Disease; Humans; Infant; Ti | 2020 |
Dual-action ambroxol in treatment of chronic pain in Gaucher Disease.
Topics: Adult; Ambroxol; Analgesics; Chronic Pain; Female; Gaucher Disease; Humans; Quality of Life | 2020 |
Upgrading the evidence for the use of ambroxol in Gaucher disease and GBA related Parkinson: Investigator initiated registry based on real life data.
Topics: Adolescent; Adult; Aged; Ambroxol; Biological Availability; Blood-Brain Barrier; Child; Child, Presc | 2021 |
Ambroxol increases glucocerebrosidase (GCase) activity and restores GCase translocation in primary patient-derived macrophages in Gaucher disease and Parkinsonism.
Topics: Adult; Aged; Aged, 80 and over; Ambroxol; Cells, Cultured; Enzyme Inhibitors; Female; Gaucher Diseas | 2021 |
Early initiation of ambroxol treatment diminishes neurological manifestations of type 3 Gaucher disease: A long-term outcome of two siblings.
Topics: Ambroxol; Child; Child, Preschool; Enzyme Replacement Therapy; Female; Gaucher Disease; Glucosylcera | 2021 |
Transgenic mice expressing human glucocerebrosidase variants: utility for the study of Gaucher disease.
Topics: Ambroxol; Animals; Cerebellum; Cerebral Cortex; Disease Models, Animal; Enzyme Activation; Female; G | 2013 |
Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells.
Topics: Adult; Aged; Aged, 80 and over; Ambroxol; Cells, Cultured; Female; Fibroblasts; Gaucher Disease; Gen | 2014 |
Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells.
Topics: Adult; Aged; Aged, 80 and over; Ambroxol; Cells, Cultured; Female; Fibroblasts; Gaucher Disease; Gen | 2014 |
Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells.
Topics: Adult; Aged; Aged, 80 and over; Ambroxol; Cells, Cultured; Female; Fibroblasts; Gaucher Disease; Gen | 2014 |
Ambroxol improves lysosomal biochemistry in glucocerebrosidase mutation-linked Parkinson disease cells.
Topics: Adult; Aged; Aged, 80 and over; Ambroxol; Cells, Cultured; Female; Fibroblasts; Gaucher Disease; Gen | 2014 |
Magic shotgun for Parkinson's disease?
Topics: alpha-Synuclein; Ambroxol; Female; Fibroblasts; Gaucher Disease; Glucosylceramidase; Humans; Male; M | 2014 |
The contribution of mutant GBA to the development of Parkinson disease in Drosophila.
Topics: Ambroxol; Animals; Disease Models, Animal; Dopaminergic Neurons; Drosophila melanogaster; Endoplasmi | 2016 |
Ambroxol effects in glucocerebrosidase and α-synuclein transgenic mice.
Topics: alpha-Synuclein; Ambroxol; Animals; Brain; Disease Models, Animal; Expectorants; Gaucher Disease; Gl | 2016 |
The additive effect on the antiepileptic treatment of ambroxol in type 3 Gaucher patient. The early observation.
Topics: Adult; Ambroxol; Anticonvulsants; Drug Resistant Epilepsy; Expectorants; Female; Gaucher Disease; Hu | 2018 |
Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease.
Topics: Ambroxol; Amino Acid Sequence; Binding Sites; Catalytic Domain; Cells, Cultured; Drug Evaluation, Pr | 2009 |
Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease.
Topics: Ambroxol; Amino Acid Sequence; Binding Sites; Catalytic Domain; Cells, Cultured; Drug Evaluation, Pr | 2009 |
Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease.
Topics: Ambroxol; Amino Acid Sequence; Binding Sites; Catalytic Domain; Cells, Cultured; Drug Evaluation, Pr | 2009 |
Identification and characterization of ambroxol as an enzyme enhancement agent for Gaucher disease.
Topics: Ambroxol; Amino Acid Sequence; Binding Sites; Catalytic Domain; Cells, Cultured; Drug Evaluation, Pr | 2009 |
The chaperone activity and toxicity of ambroxol on Gaucher cells and normal mice.
Topics: Ambroxol; Animals; beta-Glucosidase; Body Weight; Cells, Cultured; Colorimetry; Dose-Response Relati | 2013 |
The chaperone activity and toxicity of ambroxol on Gaucher cells and normal mice.
Topics: Ambroxol; Animals; beta-Glucosidase; Body Weight; Cells, Cultured; Colorimetry; Dose-Response Relati | 2013 |
The chaperone activity and toxicity of ambroxol on Gaucher cells and normal mice.
Topics: Ambroxol; Animals; beta-Glucosidase; Body Weight; Cells, Cultured; Colorimetry; Dose-Response Relati | 2013 |
The chaperone activity and toxicity of ambroxol on Gaucher cells and normal mice.
Topics: Ambroxol; Animals; beta-Glucosidase; Body Weight; Cells, Cultured; Colorimetry; Dose-Response Relati | 2013 |
Commentary on "Pilot study using ambroxol as a pharmacological chaperone in type 1 Gaucher disease" by Zimran et al.
Topics: Ambroxol; Female; Gaucher Disease; Humans; Male | 2013 |
Ambroxol as a pharmacological chaperone for mutant glucocerebrosidase.
Topics: Ambroxol; Cells, Cultured; Combined Modality Therapy; Dose-Response Relationship, Drug; Drug Evaluat | 2013 |