ambroxol oral / Generic mfg. - LARVOL DELTA

Safety and Efficacy of Ambroxol Therapy in Polish Patients with Gaucher Disease. (PubMed, Life (Basel)) - "In patients with type 3 GD receiving stable ERT, ambroxol demonstrated beneficial effects on neurological symptom expression. Some improvement was observed in biomarkers; the activity of chitotrosidase and concentration of lyso-Gl1 decreased. These findings support the therapeutic potential of ambroxol as an adjunctive treatment for neuronopathic Gaucher disease."

Journal • Gaucher Disease • Genetic Disorders • Hematological Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Local therapeutic strategies for neurocutaneous dysesthesia: from capsaicin to cannabinoids. (PubMed, J Dermatolog Treat) - "The following local treatment options are described: capsaicin, calcineurin inhibitors, menthol, pramoxine/lidocaine/prilocaine, ambroxol, phenytoin, strontium, amitriptyline, doxepin, duloxetine, baclofen, gabapentin, cannabinoids, loperamide, naltrexone, clonidine, prazosin, acetylsalicylic acid, diclofenac, aprepitant, ketamine, calamine, polidocanol, and botulinum toxin type A. Local therapy offers a valuable and often well-tolerated first step in managing ND...Clinicians are advised to start with low concentrations, discuss off-label use, and monitor for side effects. Until robust guidelines are available, a cautious trial-and-error approach remains the cornerstone of management."

Journal • Review • Dermatology • Neuralgia • Pain • Pruritus

INVESTIGATING NOVEL BIOMARKERS IN PRE-CLINICAL MODELS OF PARKINSON'S DISEASE (ADPD 2026) - "We validated these findings with a compound known to enhance GBA activity. Primary rat dopaminergic neurons were injured with α-syn PFF and conduritol B epoxide (CBE; GBA inhibitor; 20 µM) +/- Ambroxol (500 nM)... In conclusion, we have shown that our in-vitro and in-vivo models of PD are useful, translatable tools to detect biochemical changes that can serve as biomarkers of disease progression. These results support the development of novel biomarkers for PD to improve disease detection and early intervention."

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

UPDATE ON THE GRONINGEN EARLY-PD AMBROXOL TREATMENT (GREAT) TRIAL: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, SINGLE CENTER TRIAL WITH AMBROXOL IN PARKINSON PATIENTS WITH A GBA MUTATION (ADPD 2026) - "1. Ambroxol up to 1800 mg per day is well-tolerated 2. The currently included GBA mutations are mainly risk variants"

Clinical • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

AMBROXOL DISPLACES ALPHA-SYNUCLEIN FROM THE MEMBRANE AND INHIBITS THE FORMATION OF EARLY PROTEIN-LIPID COAGGREGATES (ADPD 2026) - "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 αS into amyloid fibrils."

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

PRE-RECORDED: THE EFFECTS OF 78-WEEK TREATMENT WITH AMBROXOL ON COGNITIVE, MOTOR, AND NEUROPSYCHIATRIC SYMPTOMS IN PARKINSON'S DISEASE DEMENTIA (ADPD 2026) - P2 | "Ambroxol had an overall positive effect on neuropsychiatric symptoms over 78 weeks, whereas only GBA1 mutation carriers seemed to show cognitive benefit. Safety and pharmacology in the OLE were similar to the double-blind phase."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • Movement Disorders • Parkinson's Disease • Psychiatry • GBA • GBA1

NL76160.018.21: Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series (clinicaltrialsregister.eu) - P2/3 | N=4 | Recruiting | Sponsor: Amsterdam UMC Stichting | Not yet recruiting ➔ Recruiting

Enrollment open • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Non-Pulmonary Mechanisms of Action of Ambroxol in In Vivo Experimental Models: a Systematic Review. (PubMed, Fundam Clin Pharmacol) - "Despite ambroxol's widespread clinical use, limited research has explored its non-respiratory applications. Existing studies suggest its promising therapeutic potential, reinforcing the need for further investigation into its role as an alternative treatment for various inflammatory and oxidative stress-related conditions beyond pulmonary diseases."

Journal • Preclinical • Review • Cardiovascular • CNS Disorders • Dermatitis • Dermatology • Hematological Disorders • Immunology • Inflammation • Neuralgia • Pain • Psoriasis • Psychiatry • Pulmonary Disease • Reperfusion Injury • Respiratory Diseases • IL1B • TNFA

GREAT: GRoningen Early-PD Ambroxol Treatment (clinicaltrials.gov) - P2/3 | N=80 | Recruiting | Sponsor: University Medical Center Groningen | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

Network meta-analysis of pharmacological treatments for idiopathic pulmonary fibrosis: evaluating effects on lung function. (PubMed, Front Pharmacol) - "N-acetylcysteine (NAC) combined with Roxithromycin (RXM) was the most effective intervention for improving Vital Capacity (VC) (SUCRA: 88.8%) and Forced Expiratory Volume in 1 s/Forced Vital Capacity (FEV1/FVC) (SUCRA: 97.45%). Ambroxol was the most effective intervention for improving Total Lung Capacity (TLC) (SUCRA: 82.52%), while Thalidomide was the most effective intervention for improving Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) (SUCRA: 90.93%)...Additionally, future research should examine the long-term effectiveness of new drugs like Nerandomilast and Pamrevlumab, while also improving comprehensive assessments of synergistic changes across various pulmonary function indicators. https://www.crd.york.ac.uk/PROSPERO/view/CRD420251148658."

Journal • Retrospective data • Review • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases

Efficacy of ambroxol hydrochloride and clenbuterol hydrochloride oral solution, its influencing factors, and its effect on quality of life in child patients with lower respiratory tract infections and symptoms of mucoid sputum. (PubMed, Respir Med) - "AHCHOS alleviates respiratory symptoms and improves quality of life with good safety in child patients with LRTI and symptoms of mucoid sputum, which is more beneficial for those with elevated respiratory rate, higher neutrophil percentage, and nonparental guardians."

HEOR • Journal • Cough • Infectious Disease • Respiratory Diseases

Comparative efficacy of tympanostomy tube insertion for secretory otitis media following radiotherapy for head and neck malignancies: a network meta-analysis. (PubMed, Acta Otolaryngol) - "TI performed with preinsertion ambroxol or triamcinolone was associated with a reduced recurrence rate...ITI and TC were associated with higher complication risks. Key limitations include short follow-up duration for LT and the predominance of unblinded study designs."

Journal • Retrospective data • Head and Neck Cancer • Nasopharyngeal Carcinoma • Oncology • Otorhinolaryngology • Solid Tumor

Leveraging Hafnium-Doped Tungsten Oxide as an Ultrasensitive Electrochemical Tool for the Detection of Ambroxol. (PubMed, Chembiochem) - "Reasonable recovery rates from pharmaceutical and urine samples showed the accuracy and reliability of the sensor for real-world sample analysis. The proposed work is promising in quantifying ambroxol at trace levels, representing a cost-effective and a direct method for clinical analysis and pharmaceutical quantification."

Journal

Stability study of pharmacy compounded high-dose ambroxol hydrochloride capsules for an n-of-1 clinical trial involving Dutch patients with Gaucher disease type 3. (PubMed, Eur J Hosp Pharm) - "The 75 mg ambroxol HCl capsules are stable for at least 6 months at room temperature. This paper provides guidance to pharmacies for compounding of high-dose ambroxol HCl capsules to ensure the availability of ambroxol for patients in need."

Journal • Gaucher Disease • Genetic Disorders • Metabolic Disorders

Ambroxol for Parkinson's Disease. (PubMed, Mov Disord) - No abstract available

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

Expert opinion and clinical practice recommendations using a modified Delphi approach for pediatric acute cough evaluation and management in India. (PubMed, Lung India) - "The present consensus fills the existing need and will guide family physicians in successfully diagnosing and managing cough in the primary healthcare setting in India."

Journal • Allergy • Cough • Immunology • Infectious Disease • Pediatrics • Pulmonary Disease • Respiratory Diseases

Protocol of ASPro-PD: a phase 3 trial of ambroxol to slow progression in genetically stratified Parkinson's disease. (PubMed, J Neurol) - "This trial is the largest to date to study the effect of ambroxol in PD, utilise a genetically stratified PD population and will provide robust estimates of the efficacy of ambroxol in slowing PD clinical progression."

Journal • P3 data • CNS Disorders • Movement Disorders • Parkinson's Disease • Respiratory Diseases • GBA1

Mechanical ventilation strategies and lung protection during cardiopulmonary bypass: A scoping review. (PubMed, Perfusion) - "When combined with protective measures (low tidal volume [LTV], moderate positive end-expiratory pressure [PEEP], and recruitment maneuvers), they show clinical efficacy in lung protection. Pharmacologic agents like ambroxol, neutrophil elastase inhibitors, and sivelestat further enhance protection, underscoring the promise of combined mechanical-pharmacological strategies against CPB-induced lung injury.ConclusionsA comprehensive approach integrating individualized ventilation management and pharmacological intervention is key to reducing CPB-induced lung injury, enhancing postoperative respiratory function, and improving patient outcomes."

Journal • Review • Cardiovascular • Inflammation • Respiratory Diseases • ELANE

Exploring the long-term use of ambroxol in Gaucher disease type 2: insights from two pediatric cases. (PubMed, Front Neurol) - "Neurocognitive and motor function assessments at 40 months of age indicated a moderate to severe developmental delay, yet continuous developmental progress. These interim findings contribute to the mounting evidence supporting ABX as a variant-dependent treatment for GD2 patients."

Journal • Developmental Disorders • Gaucher Disease • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics • Rare Diseases

The protective effect of ambroxol against cyclophosphamide-induced acute kidney injury involves Nrf2/HO-1 signaling upregulation and suppression of oxidative and inflammatory damage. (PubMed, Naunyn Schmiedebergs Arch Pharmacol) - "In conclusion, these findings suggest that ABX confers nephroprotection against CP-induced injury primarily through its antioxidant, anti-inflammatory, and anti-apoptotic actions, partly via modulation of the Keap-1/Nrf2/HO-1 pathway. These findings support potential repurposing of ABX as a nephroprotective adjuvant during CP chemotherapy and warrant further clinical investigation."

Journal • Acute Kidney Injury • Inflammation • Nephrology • Renal Disease • CASP3 • CAT • IL1B • KEAP1 • KIM1 • TNFA

Comparative efficacy of doxophylline-ambroxol combination versus aminophylline in the management of acute exacerbations of COPD: A retrospective case-control study. (PubMed, Pak J Pharm Sci) - "Doxophylline-ambroxol combination therapy is more effective than aminophylline in improving pulmonary function and reducing inflammation in AECOPD, with equivalent safety."

Clinical • Journal • Retrospective data • Chronic Obstructive Pulmonary Disease • Immunology • Inflammation • Pulmonary Disease • Respiratory Diseases

Clinical Application of BAL Combined with Microscopic Spraying Inhalation of Sputum Mucus Lysate in the Treatment of Non asthmatic and Refractory Pneumonia in Children (ChiCTR) - P=N/A | N=108 | Completed | Sponsor: Guangzhou Panyu District Central Hospital; Guangzhou Panyu District Central Hospital

New trial • Asthma • Infectious Disease • Pneumonia • Respiratory Diseases

Drug-Induced Liver Injury with Eosinophilia: A Case-Control Study Using Electronic Medical Records. (PubMed, Biol Pharm Bull) - "Sulbactam/cefoperazone showed the strongest association with DILI-Eos (adjusted odds ratio (OR) 14.51; 95% confidence interval (CI) 10.09-20.85), followed by meropenem (OR 5.68; 95% CI 4.10-7.82) and tazobactam/piperacillin (OR 3.55; 95% CI 2.63-4.75). Several commonly used drugs, such as mosapride, lansoprazole, furosemide, and ambroxol, were also significantly associated with increased risk...Notably, substantial variability in risk was observed even within the same drug classes, such as β-lactam antibiotics and nonsteroidal anti-inflammatory drugs (NSAIDs), underscoring the importance of drug-specific evaluation. Further studies are needed to clarify the causality and mechanisms underlying eosinophilic responses in DILI."

Journal • Eosinophilia • Hepatology • Immunology • Liver Failure

First use of nitazoxanide in Kelleni's protocol for managing severe bronchiolitis in a 9-month-old infant: A case report and review of literature. (PubMed, World J Clin Cases) - "To the best of my knowledge, this report presents, for the first time globally, a potential of nitazoxanide within Kelleni's protocol to early manage infants younger than one year suffering from severe lower respiratory tract viral infection at home. Selective antitussive treatment, using agents such as low dose benproperine, was employed to mitigate troublesome cough and improve patient comfort without compromising respiratory function. Additionally, alpha amylase was used to facilitate pulmonary secretion clearance. The protocol aims to reduce morbidity and mortality from viral lower respiratory tract infections in this vulnerable population."

Journal • Cough • Infectious Disease • Inflammation • Novel Coronavirus Disease • Pulmonary Disease • Respiratory Diseases

Can We Develop Glioma Subtype-Specific Precision Medicines? An Integrative Machine Learning Pipeline for Biomarker Discovery and Drug Repurposing for Glioblastoma and Low-Grade Glioma. (PubMed, OMICS) - "Repurposed drug candidates were then predicted via signature-based prioritization and evaluated using molecular docking simulations, revealing six promising compounds for GBM (vandetanib, capecitabine, melatonin, agomelatine, ramelteon, and tasimelteon) and one for LGG (ambroxol). This study demonstrates the utility of combining class-balancing, feature selection, and drug repurposing pipelines to uncover clinically relevant glioma biomarkers and therapeutic candidates, thus providing a computational foundation for future experimental and translational validation in these brain cancers and neuro-oncology."

Biomarker • Journal • Brain Cancer • Glioblastoma • Glioma • Oncology • Solid Tumor • ANK3