ambroxol oral / Generic mfg. - LARVOL DELTA

Long-term oral glucocerebrosidase activator reduces soluble α-synuclein oligomer accumulation in Parkinsonian LRRK2 mutant mouse brain. (PubMed, NPJ Parkinsons Dis) - "Here, we showed that long-term treatment of ambroxol (ABX), a brain-penetrant GCase activator, reduced α-syn oligomer accumulation in aged mutant LRRK2R1441G mouse striatum...This regimen significantly reduced α-syn oligomer level in mutant striatum to a comparable physiological level in age-matched WT without altering total α-syn and Ser129-phosphorylation levels. This is the first study demonstrating reduced α-syn oligomer accumulation by chronic treatment of GCase activator in aged mouse brains vulnerable to PD, suggesting early intervention to alter progression of synucleinopathies as a key determinant of clinical outcomes of PD."

Journal • Preclinical • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • LRRK2

Modest improvement of metabolic and behavioral deficits with long-term ambroxol treatment in a Pink1-/-SNCAA53T double mutant mouse model of Parkinson's disease. (PubMed, Acta Pharmacol Sin) - "In addition, its lysosomal accumulation may further disrupt sphingolipid metabolism and impair mitochondrial compensatory mechanisms. Ambroxol-induced lysosomal exocytosis may transiently relieve αSyn burden, but further interventions would be required to ensure αSyn clearance from the brain."

Journal • Preclinical • Alzheimer's Disease • CNS Disorders • Dementia • Metabolic Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

The use of genistein and ambroxol may be an effective approach in correcting cellular dysfunctions of mucopolysaccharidosis-plus syndrome. (PubMed, Mamm Genome) - "Using a line of MPSPS patient-derived fibroblasts, we demonstrated that treatment with genistein and ambroxol resulted in elevation of the mutant VPS33A protein level, as well as in improvement or correction of various previously reported cellular defects, including GAG levels, endosomal markers, and cytoskeleton elements. In the light of these results, and since both genistein and ambroxol were previously demonstrated to be safe when used in relatively high amounts, we suggest that the use of these compounds, and especially their combination, might be considered as a potential therapeutic approach in MPSPS, which is currently an incurable disease."

Journal • Metabolic Disorders • Targeted Protein Degradation

Ambroxol displaces α-synuclein from the membrane and inhibits the formation of early protein-lipid coaggregates. (PubMed, Chem Sci) - "We find that Ambroxol not only displaces α-synuclein from negatively charged membranes but also prevents the formation of early α-synuclein-lipid coaggregates during primary nucleation. These results suggest that Ambroxol may have beneficial effects on other synucleinopathies, such as multiple system atrophy and dementia with Lewy Bodies, that are also characterised by the aggregation of α-synuclein into amyloid fibrils."

Journal • CNS Disorders • Dementia • Lewy Body Disease • Movement Disorders • Multiple System Atrophy • Parkinson's Disease • GBA

Ambroxol's Role in PTZ-Induced Anxiety-Like Conditions in Mice: Targeting Neuroinflammation and Oxidative Stress via In Vivo and In Silico Approaches. (PubMed, Mol Neurobiol) - "Ambroxol exhibited anxiolytic efficacy comparable to diazepam, with no apparent sedative effects observed behaviorally, although direct assessment of sedation (e.g., rotarod test) was not performed. Ambroxol demonstrated promising multi-target neuroprotective potential by concurrently attenuating oxidative and inflammatory cascades, supporting its further exploration as a safe, repurposed therapeutic candidate for anxiety disorders."

Journal • Preclinical • Anesthesia • CNS Disorders • Epilepsy • Inflammation • Mental Retardation • Mood Disorders • Psychiatry • IL1B • TNFA

Analysis of Solubility, pH, Antimicrobial Action and Cytotoxicity of Calcium Hydroxide Paste Associated With Ambroxol Hydrochloride. (PubMed, Aust Endod J) - "The association did not alter CH's cytotoxicity. Further research is needed for optimal ambroxol concentration."

Journal

Ambroxol: mechanisms of action and efficacy for inhaled therapy in the treatment of respiratory diseases. (PubMed, Recenti Prog Med) - "Moreover, ambroxol stimulates the synthesis of pulmonary surfactant, which, in addition to increasing lung compliance, preventing end-expiratory atelectasis, and facilitating the recruitment of collapsed airways, has anti-inflammatory, anti-infective, and immunoregulatory properties. Ambroxol inhalation represents an efficient way to reduce sputum viscosity, increase expectoration without increasing adverse events."

Journal • Pulmonary Disease • Respiratory Diseases

Exploring Novel Therapeutic Avenues: Drug Repurposing for Neurodegenerative Movement Disorders. (PubMed, Curr Drug Res Rev) - "Several studies on the potential of pre-existing drugs such as isradipine, tetracycline, ambroxol, metformin, deferiprone, simvastatin, etc., which have been repurposed for neurodegenerative movement disorders, including Parkinson's disease, Huntington's disease, Alzheimer's disease, Multiple Sclerosis, etc. have been discussed. Further, the current scenario and future prospective of drug repurposing have also been touched upon."

Journal • Review • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders • Huntington's Disease • Movement Disorders • Multiple Sclerosis • Parkinson's Disease

Perinatal-onset neuronopathic Gaucher disease is refractory to high-dose ambroxol: A case report and literature review. (PubMed, Neonatology) - "Ambroxol therapy may be insufficient to improve the prognosis of patients with PnGD, underscoring the limitations of early intervention in newborn-screened patients with GD. Therefore, pre-emptive therapeutic strategies are required to rescue and cure neonates with PnGD."

Journal • Cardiovascular • CNS Disorders • Congestive Heart Failure • Epilepsy • Gaucher Disease • Genetic Disorders • Heart Failure • Hematological Disorders • Hepatology • Liver Failure • Metabolic Disorders

The development of ambroxol in als/mnd (ALS-MND 2025) - P2, P3 | "The role of patient associations could be critical depending on the business model applied, and any development spin-out configuration. These issues are typical of development of generic drugs for rare diseases and merit discussion."

Alzheimer's Disease • CNS Disorders • Dementia • Gaucher Disease • Genetic Disorders • Lewy Body Disease • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Rare Diseases • Type 1 Gaucher Disease

Does Amyotrophic Lateral Sclerosis (ALS) Have Metabolic Causes from Human Evolution? (PubMed, Cells) - "A GBA2 inhibitor, ambroxol, is currently in phase II for ALS...Lipid/lactate metabolism changed to support these evolutionary changes and lactate is a major body/brain fuel, but compromised in ALS patients and a marker of disease progression. Recent progress in sports science involving lactate metabolism and human performance may also be relevant to ALS therapies, and incidence."

Journal • Review • Amyotrophic Lateral Sclerosis • CNS Disorders • Infectious Disease

Ambroxol as adjunctive therapy for neurologic symptoms in Gaucher disease type 3: Case report. (SSIEM 2023) - No abstract available

Case report • Clinical • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Endothelial to Mesenchymal Transition in Gaucher Disease and Role of High-Dose Ambroxol (SSIEM 2023) - No abstract available

Gaucher Disease • Genetic Disorders • Metabolic Disorders

A 10-year experience with high-dose ambroxol in combination with enzyme replacement therapy for neuropathic Gaucher disease. (SSIEM 2023) - No abstract available

Combination therapy • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Endothelial to Mesenchymal Transition in Gaucher Disease and Role of High-Dose Ambroxol (SSIEM 2023) - No abstract available

Gaucher Disease • Genetic Disorders • Metabolic Disorders

A 10-year experience with high-dose ambroxol in combination with enzyme replacement therapy for neuropathic Gaucher disease. (SSIEM 2023) - No abstract available

Combination therapy • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Endothelial to Mesenchymal Transition in Gaucher Disease and Role of High-Dose Ambroxol (SSIEM 2023) - No abstract available

Gaucher Disease • Genetic Disorders • Metabolic Disorders

A 10-year experience with high-dose ambroxol in combination with enzyme replacement therapy for neuropathic Gaucher disease. (SSIEM 2023) - No abstract available

Combination therapy • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Endothelial to Mesenchymal Transition in Gaucher Disease and Role of High-Dose Ambroxol (SSIEM 2023) - "As a further study, we established various GBA mutant constructs for in vitro analysis under high-dose ambroxol treatment, showing increased GBA activity and decreased EMT markers in these mutant-carrying cells. CONCLUSION Our study indicates that the complex molecular process underlying ERT unresponsiveness in GD and high- dose ambroxol might have a role in relieving this process."

Gaucher Disease • Genetic Disorders • Hematological Malignancies • Lymphoma • Metabolic Disorders • Multiple Myeloma • Oncology

Ten-Year Experience with High-Dose Ambroxol in Combination with Enzyme Replacement Therapy for Neuropathic Gaucher Disease (SSIEM 2023) - "Early treatment with ambroxol, rather than delayed treatment, seems to be associated with better long-term outcomes in nGD patients."

Combination therapy • CNS Disorders • Epilepsy • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Ambroxol rescues cognitive impairment by ameliorating oxidative stress and autophagic dysfunction in the streptozotocin-induced Alzheimer's disease model. (PubMed, Eur J Pharmacol) - "It also attenuates inflammatory and autophagic dysfunction, facilitating the clearance of amyloid beta (Aβ) aggregates in the hippocampus of ICV-STZ rats. The findings of this study suggest that ambroxol could be a viable repurposed drug candidate for AD treatment."

Journal • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • Inflammation • HMOX1

Ambroxol is Effective for Corneal Pain in Rats. (PubMed, J Ocul Pharmacol Ther) - "Sparing certain CS could translate into more protection to the ocular surface. No overt toxicity of ambroxol was seen in cultured human corneal epithelial cells in this study."

Journal • Preclinical • Neuralgia • Pain • NAV1

Effect of bronchoalveolar lavage combined with high-dose ambroxol hydrochloride on severe pneumonia with respiratory failure. (PubMed, Am J Transl Res) - "The BAL + HD-AH regimen is both safe and effective for elderly SP + RF patients, accelerating rehabilitation, reducing inflammation, improving pulmonary function and respiratory mechanics, optimizing blood gas parameters, and lowering MODS and mortality risks."

Journal • Geriatric Disorders • Infectious Disease • Inflammation • Pneumonia • Respiratory Diseases

Association between early administration of mucoactive agents and in-hospital mortality in patients with pneumonia requiring mechanical ventilation: a nationwide cohort study. (PubMed, J Intensive Care) - "In patients with ventilated pneumonia, early administration of mucoactive agents was associated with lower in-hospital mortality."

Journal • Infectious Disease • Pneumonia • Respiratory Diseases

Case Report: Clinical case analysis of gaucher disease management in a resource-limited setting: a single center experience from Kashigar, Xinjiang Uygur Autonomous Region, the Western China. (PubMed, Front Pediatr) - "Treatment with high-dose ambroxol and imiglucerase significantly alleviated the patient's symptoms, and continuous follow-up was conducted to assess long-term efficacy...This has further driven interactions in various aspects such as academic exchanges, teleconsultations, and medical assistance. In turn, this has provided a solid foundation for safeguarding patients' rights and improving medical services."

Journal • Gaucher Disease • Genetic Disorders • Hematological Disorders • Metabolic Disorders • Pain • Thrombocytopenia • Type 1 Gaucher Disease