ambroxol oral / Generic mfg. - LARVOL DELTA

Ambroxol in Gaucher disease and GBA1-related Parkinson disease: an updated systematic review. (PubMed, Acta Neurol Belg) - "Biomarker changes alone do not establish clinical efficacy. ABX remains an off-label, investigational treatment in GD and GBA1-PD, and no GBA1 genotype or functional assay currently defines a validated indication."

Journal • Review • CNS Disorders • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Movement Disorders • Parkinson's Disease

Small-Molecule NANT Therapeutics Targeting the Brain-Immune Axis in Alzheimer's Disease: Mechanisms, Clinical Progress, and Translational Challenges. (PubMed, Molecules) - "We reviewed several agents that affect the brain/immune axis, namely, kinase inhibitors (nelflamaimod and masitinib), NLRP3 inflammasome inhibitors (MCC950, selnoflast, and dapansutrile), TREM2 activators (VG-3927), gingipain inhibitors, PPARγ activators, KCa3.1 inhibitors, and repurposed drugs such as ambroxol and cromolyn. Additional translational challenges included sex-specific differences in neuroimmune responses, APOE ε4-associated immune dysfunction, and the need for biomarker-guided patient selection. Although immune-targeted therapies remain strongly supported by biological evidence in AD, meaningful clinical progress will likely require earlier intervention strategies guided by biomarkers and therapies capable of targeting multiple disease pathways simultaneously."

Journal • Review • Alzheimer's Disease • CNS Disorders • Inflammation • APOE • NLRP3 • PPARG

From engagement to dissemination: a WiseAge patient and public involvement and engagement event on dementia with Lewy bodies. (PubMed, Front Dement) - "This study explored how attendees evaluated information about dementia with Lewy bodies and the ongoing Ambroxol in New and Early Dementia with Lewy Bodies (ANeED) clinical drug trial presented at a WiseAge public engagement event, and how they reported sharing this information afterwards...The event was well received and supported secondary dissemination through attendees' interpersonal networks, particularly friends and family. Although no immediate recruitment effect was observed, public engagement may extend the reach of dementia research communication beyond those who attend."

Journal • Alzheimer's Disease • CNS Disorders • Dementia • Lewy Body Disease

Rebalancing α-Synuclein Clearance: Novel Therapeutic Frontiers in Parkinson's Disease. (PubMed, Neuromolecular Med) - "Potential treatment modalities include small molecules targeting lysosome function (ambroxol, rapamycin, TFEB activators), CMA enhancers, gene therapy, and antibodies against extracellular α-synuclein. Furthermore, new modalities like molecular glue degraders, allostery-based stabilization of α-synuclein tetramers, engineered decoy particles, and bispecific antibodies represent some other possible routes towards multimodal disease modification."

Journal • Review • CNS Disorders • Gene Therapies • Inflammation • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Targeted Protein Degradation • TFEB

Ambroxol as an uncommon trigger of non-pigmenting fixed drug eruption: a case report (EADV 2026) - No abstract available

Case report • Clinical

SBK001: A Novel Multi-Target Candidate Drug for Protecting Against Acute Lung Injury - Preclinical Pharmacodynamic Evidence from Rodent Models (ERS 2026) - "Conclusion SBK001 exhibits significant protective effects on both ALI models, improving lung pathological damage via multiple mechanisms, reducing mortality, and showing a clear dose-dependent response. Its efficacy is superior to ambroxol hydrochloride,providing strong preclinical evidence for the development of new ALI drugs."

PK/PD data • Preclinical • Acute Lung Injury • Respiratory Diseases

Therapeutics candidates and repurposing strategies to target parkinson's disease pathology: current evidence and future directions. (PubMed, Metab Brain Dis) - "For proteostatic dysfunction, the USP14 inhibitor IU1 enhanced both proteasomal and autophagic flux, though neurotoxicity above 200 µM limits its therapeutic window; BIIB122, a selective LRRK2 inhibitor, achieved CNS target engagement in Phase 1/2 trials, but its Phase 3 LIGHTHOUSE study was terminated, with Phase 2b LUMA ongoing. In dopaminergic neurodegeneration, GLP-1 receptor agonists produced divergent outcomes: Exenatide-PD3 (n = 231) showed no benefit over placebo, whereas LixiPark demonstrated reduced motor progression in early-stage PD, revealing that mechanistic equivalence does not guarantee clinical equivalence across heterogeneous patient populations...Repurposed compounds, including ambroxol, N-acetylcysteine, doxycycline, metformin, and GLP-1 receptor agonists, demonstrate engagement with disease-relevant mechanisms and, in several cases, preliminary evidence of clinical activity...Future priorities must include: (i) precision stratification by..."

Journal • Review • CNS Disorders • Inflammation • Metabolic Disorders • Movement Disorders • Parkinson's Disease • GBA • IL1B • LRRK2 • MMP3 • MMP9 • TNFA • USP14

High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3) (clinicaltrials.gov) - P=N/A | N=12 | Completed | Sponsor: Agyany Pharma LTD | Terminated ➔ Completed

Trial completion • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Pediatrics • Type 1 Gaucher Disease

Glucocerebrosidase dysfunction in GBA1 carriers: insights from blood and macrophage analyses. (PubMed, Mol Biol Rep) - "Biochemical alterations in GCase function are linked to GBA1 mutations independent of PD status, with "severe" mutations notably impacting lysosomal function and multiple cellular processes. We propose the GCase activity to HexSph ratio as a novel biomarker for disease progression and monitoring therapeutic response."

Journal • CNS Disorders • Movement Disorders • Parkinson's Disease • CTSD • GBA • GBA1

Analysis of the interventional effects of ambroxol hydrochloride and ontelukast sodium combined with azithromycin on immune function and C-reactive protein serum expression in children with severe Mycoplasma pneumoniae pneumonia. (PubMed, Front Pediatr) - "The triple combination of ambroxol hydrochloride, montelukast sodium, and azithromycin is associated with improved inflammatory status, enhanced immune function, and better pulmonary function in children with SMPP, with a favorable safety profile. These findings warrant confirmation in prospective, multicenter randomized controlled trials."

Journal • Cough • Infectious Disease • Oncology • Pneumonia • Respiratory Diseases • CD4 • CD8 • CRP • IFNG • IL2 • IL4 • TNFA

Chemical tools for inhibition and activity-based profiling of glucocerebrosidase in vitro and in vivo. (PubMed, Cell Chem Biol) - "Robust characterization shows that compound CAz-5c selectively inactivates GCase in vitro, in live cells, and in vivo-including GCase in the brains of live mice, at low doses, overcoming a limitation of previously reported inhibitors. Collectively, these tools can be used to generate titratable chemical models of neuropathic GCase deficiency in animals with various genetic backgrounds with unrivaled molecular precision."

Journal • Preclinical • CNS Disorders • Gaucher Disease • Genetic Disorders • Metabolic Disorders • Movement Disorders • Parkinson's Disease

Association of Ambroxol Hydrochloride and Clenbuterol Hydrochloride Oral Solution with Respiratory Symptom Improvement and Safety in Chinese Children with Pneumonia: A Real-World Propensity Score-Matched Study. (PubMed, Infect Drug Resist) - P=N/A | "These findings should be interpreted as observational and hypothesis-generating. The study was registered at Chinese Clinical Trial Registry (https://www.chictr.org.cn/, ChiCTR1800015818)."

Journal • Real-world evidence • Cough • Infectious Disease • Pneumonia • Respiratory Diseases

TAME-CLC: Muco-active Agent Use and Clinical Outcomes After Lung Cancer Diagnosis in Chronic Obstructive Pulmonary Disease (clinicaltrials.gov) - P=N/A | N=27000 | Active, not recruiting | Sponsor: Seoul National University

New trial • Chronic Obstructive Pulmonary Disease • Immunology • Lung Cancer • Oncology • Pulmonary Disease • Respiratory Diseases • Solid Tumor

Efficacy and safety of inhaled ambroxol hydrochloride solution in Chinese pediatric patients with acute lower respiratory tract infections: a real-world, multicenter, open-label, single-arm study. (PubMed, Transl Pediatr) - "While the single-arm design limits causal inference, these findings provide robust real-world evidence supporting AHSI as an effective expectorant option. Prospective confirmation through RCTs will further define its role in first-line therapy."

Journal • Real-world evidence • Asthma • Cough • Hematological Disorders • Immunology • Infectious Disease • Interstitial Lung Disease • Liver Failure • Nephrology • Pediatrics • Pneumonia • Pulmonary Disease • Renal Disease • Respiratory Diseases

A Bayesian reappraisal of disease-modifying drug candidates for Parkinson's Disease (EAN 2026) - " We performed a Bayesian reappraisal of major DMT trials—covering cinpanemab, deferiprone, CoQ10, ambroxol, NLY01, liraglutide, lixisenatide, and exenatide—focusing on motor and non-motor outcomes, with standardized effect sizes, Bayes Factors (BF₁₀), and posterior probabilities. Bayesian analysis aligns with the strongest frequentist signals while clarifying ambiguous or inconsistent results, underscoring that the magnitude of the effect and evidential strength vary substantially across compounds. GLP-1R agonists show promising efficacy as DMT candidates for PD treatment. Pharmacokinetic and pharmacodynamic differences among the compounds could underlie the different efficacy profiles concerning the disease stage and different symptoms (i.e, motor or non-motor)."

CNS Disorders • Movement Disorders • Parkinson's Disease

Photodegradation of expectorant drug ambroxol in seawater environment: Seasonal impact on a global scale. (PubMed, Water Res) - "Utilizing the global-scale photolysis model GCSOLAR, this study further indicated that near-surface environmental persistence of AMB was strictly governed by geographic latitude and seasonal dynamics, where environmental half-lives remained minimal (1.24-1.83 h) in equatorial zones while rising to 34.77 h in high-latitude regions during winter. These findings highlighted the critical role of the marine matrix and global spatiotemporal variations in collectively reshaping the transformation landscape and intensifying the ecological risks of typical emerging contaminants."

Journal • Infectious Disease • Novel Coronavirus Disease

Mucoactive agents in bronchiectasis: a systematic review and meta-analysis. (PubMed, Eur Respir Rev) - "Pooled evidence did not show a reduction in exacerbations and FEV1 % pred improvements were small. Evidence certainty was low/very low, meaning overall clinical benefit remains uncertain, highlighting the need for targeted trials of specific agents and subgroups."

Clinical • Journal • Retrospective data • Review • Bronchiectasis • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Inflammation • Pediatrics • Pulmonary Disease • Respiratory Diseases

Mycoplasma pneumoniae-induced rash and mucositis in a 5-year-old child: a case report. (PubMed, J Med Case Rep) - "We recommend that pneumonia accompanied by mucocutaneous eruptions, especially in young patients, should raise clinical suspicion of MIRM. Differential diagnosis should include EM, SJS, TEN, and Kawasaki disease."

Journal • Dermatology • Infectious Disease • Mucositis • Pain • Pneumonia • Respiratory Diseases • Steven-Johnson Syndrome • Stomatitis

AMBALS - OLE: An Open Label Extension of the double-blind randomised, placebo-controlled clinical trial to test the treatment of amyotrophic lateral sclerosis with ambroxol. (ANZCTR) - P2 | N=30 | Active, not recruiting | Sponsor: The Florey Institute of Neuroscience and Mental Health (part of the University of Melbourne) | Recruiting ➔ Active, not recruiting | N=20 ➔ 30

Enrollment change • Enrollment closed • Amyotrophic Lateral Sclerosis • CNS Disorders

AMBALS: A Double-blind Randomised, Placebo-controlled Clinical Trial to Test Ambroxol Treatment in ALS (clinicaltrials.gov) - P2 | N=50 | Completed | Sponsor: The Florey Institute of Neuroscience and Mental Health | Recruiting ➔ Completed

Trial completion • Amyotrophic Lateral Sclerosis • CNS Disorders

Association between terbutaline sulfate plus ambroxol hydrochloride and clinical outcomes, pulmonary function, and inflammatory biomarkers in elderly patients with acute exacerbation of chronic obstructive pulmonary disease. (PubMed, Front Pharmacol) - "Exploratory analysis showed a higher observed overall response rate in the combination group (92.3% vs. 75.9%, P = 0.012). In elderly patients with AECOPD, terbutaline sulfate combined with ambroxol hydrochloride was associated with greater improvements in pulmonary function, inflammatory and airway remodeling-related biomarkers, symptom relief, and length of hospital stay than terbutaline sulfate alone."

Biomarker • Clinical data • Journal • Chronic Obstructive Pulmonary Disease • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases • CXCL8 • IL6 • MMP9 • TGFB1

Ambroxol hydrochloride and clenbuterol hydrochloride oral solution versus ambroxol hydrochloride injection for pediatric lower respiratory tract infection with mucoid sputum: a multicenter, non-randomized observational study in China. (PubMed, Transl Pediatr) - "Moreover, no adverse reactions were reported in either group. AHCHOS was associated with improved respiratory symptom outcomes and was well tolerated in pediatric patients with LRTI complicated by mucoid sputum."

Journal • Observational data • Cough • Infectious Disease • Pediatrics • Pulmonary Disease • Respiratory Diseases

α-Synuclein as a molecular link between Parkinson's disease and chronic kidney disease: insights into the kidney-brain axis. (PubMed, Metab Brain Dis) - "Novel α-syn-targeted therapies, including ENT-01, Cu(II)ATSM, ambroxol, and lipid-modulating strategies, are being investigated for their cross-organ efficacy, although most evidence currently derives from preclinical or early-phase studies. Importantly, key knowledge gaps persist, including the mechanisms underlying peripheral α-syn aggregation, the pathways of inter-organ communication, and the clinical validity of α-syn-based biomarkers. Overall, current evidence supports a potential role for α-syn as a contributing molecular link between neurodegenerative and renal dysfunction, rather than a definitive unifying mechanism, underscoring the need for integrated and evidence-driven diagnostic and therapeutic approaches."

Journal • Review • Chronic Kidney Disease • CNS Disorders • Fibrosis • Immunology • Metabolic Disorders • Movement Disorders • Nephrology • Parkinson's Disease • Renal Disease • SNCA

Life-cycle exposure to tris(2-butoxyethyl) phosphate at environmentally relevant concentrations induces progressive Parkinsonian-like neurodegeneration via lysosomal dysfunction in Caenorhabditis elegans. (PubMed, Ecotoxicol Environ Saf) - "Pharmacological validation with the lysosomal chaperone ambroxol showed that improving lysosomal function mitigated TBOEP-induced neurotoxicity, supporting lysosomal dysfunction as a primary contributor to the observed pathology. Collectively, our findings identify TBOEP as a potential environmental risk factor for neurodegeneration, providing important insights that could inform further studies assessing the environmental health risks of organophosphate esters."

Journal • CNS Disorders • Movement Disorders • Parkinson's Disease

A Systematic Review on Disease-Modifying Therapies in Parkinsonian Disorders. (PubMed, Clin Pharmacol Ther) - "Glucocerebrosidase-enhancing agents, particularly ambroxol, demonstrated promising biomarker and clinical signals in early-phase trials...Repurposed drugs, including memantine and riluzole, have shown preliminary signals of benefit, though confirmatory trials are lacking...The heterogeneity of disease mechanisms and the limitations of current clinical endpoints, such as the Unified Parkinson's Disease Rating Scale, underscore the need for biomarker-driven approaches and stratified trial designs. Future success will likely depend on improved patient selection, mechanistic targeting, and the integration of fluid and imaging biomarkers to demonstrate disease modification."

Journal • Review • Alzheimer's Disease • CNS Disorders • Dementia • Lewy Body Disease • Movement Disorders • Multiple System Atrophy • Parkinson's Disease • Progressive Supranuclear Palsy