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  • ||||||||||  Plicera (afegostat tartrate) / Amicus
    [VIRTUAL] Inhibition of clostridium difficile TcdA and TcdB toxins with transition state analogues () -  Mar 28, 2021 - Abstract #ACSSp2021ACS-Sp_4402;    
    Isofagomine and noeuromycin were the most potent iminosugars identified and display uncompetitive inhibition, suggesting that both iminosugars act by binding to the enzyme-UDP complex...Finally, we confirmed that both iminosugars protected Tcd-induced rounding of IMR90 cells as visualized by light microscopy. We also showed through western blot analysis of IMR90 cell lysates that both iminosugars prevent Tcd induced toxicity by preventing the glycosylation of Rho GTPases, thereby demonstrating potential as new treatments for C. difficile.
  • ||||||||||  Galafold (migalastat) / Amicus
    Trial completion:  MAIORA: Effect of Migalastat on Cardiac Involvement in Fabry Disease (clinicaltrials.gov) -  Mar 18, 2021   
    P=N/A,  N=18, Completed, 
    We also showed through western blot analysis of IMR90 cell lysates that both iminosugars prevent Tcd induced toxicity by preventing the glycosylation of Rho GTPases, thereby demonstrating potential as new treatments for C. difficile. Active, not recruiting --> Completed
  • ||||||||||  AT-GTX-502 / Amicus
    [VIRTUAL] New Targets & Approaches to Targeting Monogenic Neurodegenerative Diseases (Track A) -  Mar 4, 2021 - Abstract #CNSS2021CNSS_6;    
    • Exploring the therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease • What is the clinical progress in rare monogenic neurodegenerative conditions? • Broadening the therapeutic landscape for ASOs in the treatment of neurodegenerative diseases
  • ||||||||||  Pombiliti (cipaglucosidase alfa-atga) / Amicus
    Enrollment closed:  A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD (clinicaltrials.gov) -  Mar 2, 2021   
    P3,  N=110, Active, not recruiting, 
    When expressed in the fly's dopaminergic cells, misfolding of α-Gal A and UPR activation led to death of these cells and to a shorter life span, which could be improved, in a mutation-dependent manner, by migalastat. Recruiting --> Active, not recruiting
  • ||||||||||  Galafold (migalastat) / Amicus
    [VIRTUAL] UNEXPECTED THICK HEARTS: A LYSOSOMAL SURPRISE (eAbstract site) -  Feb 17, 2021 - Abstract #ACC2021ACC_5114;    
    Oral migalastat was initiated. P.I232T is a rare variant that can present with isolated cardiac involvement in middle aged men with Fabry’s.
  • ||||||||||  Acomplia (rimonabant) / Sanofi, larazotide (AT1001) / 9 Meters Biopharma, Galafold (migalastat) / Amicus
    Preclinical, Journal:  Neuronal nicotinic acetylcholine receptors mediate ∆ -THC dependence: Mouse and human studies. (Pubmed Central) -  Feb 11, 2021   
    Finally, the human genetic association studies indicated that variations in the genes that code for the α5, α3, β4, and α6 nAChRs were associated with cannabis disorder phenotypes. Overall, these findings suggest that α3β4* and α6β4* nAChR subtypes represent viable targets for the development of medications to counteract THC dependence.
  • ||||||||||  Galafold (migalastat) / Amicus
    Review, Journal:  1-Deoxynojirimycin and Its Derivatives: A Mini Review of the Literature. (Pubmed Central) -  Feb 11, 2021   
    Some derivatives of 1-DNJ, like miglitol, miglustat and migalastat, were applied clinically to treat diseases such as diabetes and lysosomal storage disorders. The present review focused on the extraction, determination, pharmacokinetics and bioactivity of 1-DNJ, as well as the clinical application of 1-DNJ derivatives.
  • ||||||||||  larazotide (AT1001) / 9 Meters Biopharma
    Journal:  In silico Analysis Revealed Potential Anti-SARS-CoV-2 Main Protease Activity by the Zonulin Inhibitor Larazotide Acetate. (Pubmed Central) -  Feb 2, 2021   
    Combined, these studies suggest that AT1001, besides its well-demonstrated effect in ameliorating mucosal permeability in ALI/ARDS, may also exert a direct anti-SARS-CoV-2 effect by blocking the M. AT1001 has been used extensively in a variety of animal models of ALI demonstrating robust safety and efficacy; it is currently in phase 3 trials in celiac subjects showing strong safety and efficacy profiles. We therefore propose its use as a specific anti-SARS-CoV-2 multitargeting treatment for the current pandemic.
  • ||||||||||  AT-GTX-502 / Amicus
    Preclinical, Journal:  A tailored Cln3 mouse model for testing therapeutic interventions in CLN3 Batten disease. (Pubmed Central) -  Jan 16, 2021   
    Similar to previously characterized Cln3 mutant mouse lines, this novel model shows pathological deficits throughout the CNS including accumulation of lysosomal storage material and glial activation, and has limited perturbation in behavioral measures. Thus, at the molecular and cellular level, this mouse line provides a valuable tool for testing nonsense suppression therapies or read through compounds in CLN3 disease in the future.
  • ||||||||||  Trial initiation date:  IUERT: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (clinicaltrials.gov) -  Jan 12, 2021   
    P1,  N=10, Not yet recruiting, 
    Thus, at the molecular and cellular level, this mouse line provides a valuable tool for testing nonsense suppression therapies or read through compounds in CLN3 disease in the future. Initiation date: Dec 2020 --> Mar 2021
  • ||||||||||  Plicera (afegostat tartrate) / Amicus, venglustat (GZ402671) / Sanofi
    [VIRTUAL] NOVEL THERAPIES FOR GBA-PARKINSON DISEASE (On Demand Symposia C) -  Dec 24, 2020 - Abstract #ADPD2021ADPD_767;    
    This drug is currently in a Phase II study in GBA-PD to assess safety and target engagement, with an extension to assess impact on clinical progression over 12 months. Gene-based therapies are also in Phase II development with the intention of using CND-delivered wild-type GBA and vector to increase GCase levels and production.
  • ||||||||||  AT-GTX-502 / Amicus
    Preclinical, Journal:  Changes in motor behavior, neuropathology, and gut microbiota of a Batten disease mouse model following administration of acidified drinking water. (Pubmed Central) -  Dec 18, 2020   
    Since the gut microbiota can influence neurological functions, we examined it in our disease model and found that the gut microbiota of Cln3 mice was markedly different from control mice, and acidified water differentially changed the gut microbiota composition in these mice. These results indicate that acidified water may provide therapeutic benefit to CLN3 Batten disease patients, and that the pH of drinking water is a major environmental factor that strongly influences the results of murine behavioral and pathological studies.
  • ||||||||||  Galafold (migalastat) / Amicus
    Enrollment open:  Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial) (clinicaltrials.gov) -  Dec 3, 2020   
    P=N/A,  N=20, Recruiting, 
    These results indicate that acidified water may provide therapeutic benefit to CLN3 Batten disease patients, and that the pH of drinking water is a major environmental factor that strongly influences the results of murine behavioral and pathological studies. Not yet recruiting --> Recruiting
  • ||||||||||  Galafold (migalastat) / Amicus
    Trial completion date, Trial primary completion date, HEOR, Adherence:  MALTA-FABRY: MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study (clinicaltrials.gov) -  Nov 27, 2020   
    P=N/A,  N=30, Recruiting, 
    Migalastat can be used to treat Japanese patients with Fabry disease with GLA mutations amenable to migalastat according to the dosage and administration approved in other countries. Trial completion date: Dec 2020 --> Dec 2022 | Trial primary completion date: Dec 2019 --> Dec 2021
  • ||||||||||  AT-GTX-502 / Amicus
    Preclinical, Journal:  Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease. (Pubmed Central) -  Nov 6, 2020   
    ASOs also induced exon skipping in cell lines derived from patients with CLN3 Batten disease. Our findings demonstrate the utility of ASO-based reading-frame correction as an approach to treat CLN3 Batten disease and broaden the therapeutic landscape for ASOs in the treatment of other diseases using a similar strategy.
  • ||||||||||  Plicera (afegostat tartrate) / Amicus
    Journal:  Substituent position of iminocyclitols determines the potency and selectivity for gut microbial xenobiotic-reactivating enzymes. (Pubmed Central) -  Nov 3, 2020   
    N1 of uronic isofagomine (2) made strong electrostatic interactions with two catalytic glutamates of GUSs, resulting in the most potent inhibition (K ≥ 11 nM)...In comparison with 2, there is 200-fold enhancement in the selectivity, which was attributed to differential interactions between the propyl group and loop 5 residues of the GUSs. The results provide useful insights to develop potent and selective inhibitors for undesired GUSs.
  • ||||||||||  larazotide (AT1001) / 9 Meters Biopharma, Galafold (migalastat) / Amicus
    Preclinical, Journal:  Near-infrared spectroscopy as a tool for in vivo analysis of human muscles. (Pubmed Central) -  Oct 22, 2020   
    In addition to the non-invasive detection of tissue oxygenation, NIR spectroscopy provided the spectral signatures (ie, "fingerprints") of upper limb flexors and extensors, which represent specific, accurate, and reproducible measures of the overall biological status of these muscles. Thus, non-invasive NIR spectroscopy enables more thorough evaluation of the muscular system and optimal monitoring of the effectiveness of therapeutic or rehabilitative interventions.
  • ||||||||||  Galafold (migalastat) / Amicus
    Preclinical, Review, Journal:  In Vitro and In Vivo Amenability to Migalastat in Fabry Disease. (Pubmed Central) -  Oct 2, 2020   
    Finally, we provide a literature-based list of α-galactosidase A mutations, tested by different assays to provide a comprehensive overview of amenable mutations as a good basis for the decision-making by treating physicians. Since in vitro amenability does not always correspond with in vivo amenability, the treating clinician has the responsibility to monitor clinical and laboratory features to verify clinical response.
  • ||||||||||  Galafold (migalastat) / Amicus
    Trial completion, Trial completion date, Trial primary completion date, Monotherapy:  Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease (clinicaltrials.gov) -  Aug 9, 2020   
    P3,  N=76, Completed, 
    In the case of N370S the effectiveness in restoration of GCase activity was compatible with well-known GCase molecular chaperone IFG. Active, not recruiting --> Completed | Trial completion date: Dec 2020 --> Oct 2019 | Trial primary completion date: Dec 2020 --> Oct 2019
  • ||||||||||  AT-GAA / Amicus
    Journal:  Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease. (Pubmed Central) -  Jul 17, 2020   
    We now demonstrate in a pre-clinical study that a recently developed replacement enzyme (recombinant human GAA; AT-GAA; Amicus Therapeutics) with much improved lysosome-targeting properties reversed or significantly improved all aspects of the disease pathogenesis, an outcome not observed with the current standard of care. The therapy was initiated in GAA-deficient mice with fully developed muscle pathology but without obvious clinical symptoms; this point deserves consideration.