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  • ||||||||||  AT-GAA / Amicus, Myozyme (alglucosidase alfa) / Sanofi, miglustat / Generic mfg.
    Preclinical, Journal:  Improved efficacy of a next-generation ERT in murine Pompe disease. (Pubmed Central) -  Jun 5, 2020   
    The reversal of lysosomal and autophagic pathologies leads to improved muscle function. These data demonstrate the superiority of ATB200/AT2221 over the currently approved ERT in the murine model.
  • ||||||||||  Galafold (migalastat) / Amicus
    Review, Journal:  Current and Investigational Therapeutics for Fabry Disease. (Pubmed Central) -  Apr 11, 2020   
    However, treatment options for some patients with FD have recently expanded, with the approval of migalastat, an oral molecular chaperone...Finally, substrate reduction therapies act as inhibitors of glucosylceramide synthase, thus inhibiting the production of GB-3, promise another oral option to treat FD. This article will review the literature around current therapies as well as these newer therapeutics agents in the pipeline for FD.
  • ||||||||||  larazotide (AT1001) / Innovate Biopharma, Galafold (migalastat) / Amicus
    Journal:  Agonist Selectivity and Ion Permeation in the α3β4 Ganglionic Nicotinic Receptor. (Pubmed Central) -  Mar 31, 2020   
    The structures of the receptor in complex with nicotine, as well as the α3β4-selective ligand AT-1001, complemented by molecular dynamics, suggest principles of agonist selectivity. The structures further reveal much of the architecture of the intracellular domain, where mutagenesis experiments and simulations define residues governing ion conductance.
  • ||||||||||  Pombiliti (cipaglucosidase alfa-atga) / Amicus
    Trial completion date, Trial primary completion date:  ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (clinicaltrials.gov) -  Mar 20, 2020   
    P3,  N=14, Recruiting, 
    The structures further reveal much of the architecture of the intracellular domain, where mutagenesis experiments and simulations define residues governing ion conductance. Trial completion date: Jun 2021 --> Sep 2021 | Trial primary completion date: Feb 2021 --> Sep 2021
  • ||||||||||  AT-GTX-502 / Amicus
    Enrollment closed, Gene therapy:  Gene Therapy for Children With CLN3 Batten Disease (clinicaltrials.gov) -  Mar 12, 2020   
    P1/2,  N=7, Active, not recruiting, 
    Trial completion date: Jun 2021 --> Sep 2021 | Trial primary completion date: Feb 2021 --> Sep 2021 Recruiting --> Active, not recruiting
  • ||||||||||  AT-GTX-502 / Amicus
    An iPSC model of CLN3-Batten disease reveals a novel role of CLN3 in photoreceptor outer segment phagocytosis by RPE cells. (Exhibit Hall: Posterboard# B0044) -  Mar 9, 2020 - Abstract #ARVO2020ARVO_4464;    
    In contrast, no difference in the number of internalized POS normalized to bound POS was seen in control vs. CLN3 disease iPSC-RPE cells.Conclusions Reduced POS uptake by CLN3 disease iPSC-RPE cells is a consequence of decreased POS binding. Furthermore, these results suggest that impaired POS phagocytosis contributes to reduced accumulation of lipofuscin in CLN3-Batten disease RPE.
  • ||||||||||  Galafold (migalastat) / Amicus
    Biomarker, Journal:  Oral chaperone therapy migalastat for treating Fabry disease: enzymatic response and serum biomarker changes after one year. (Pubmed Central) -  Feb 16, 2020   
    Plasma globotriaosylsphingosine was reduced in therapy-naive patients (10.9 to 6.0 ng/mL;p=0.021), and stable (9.6 to 12.1 ng/mL;p=0.607) in patients switched from prior enzyme replacement therapy. These first real-world data show that migalastat substantially increases alpha-galactosidase-A activity, stabilizes related serum biomarkers, and improves cardiac integrity in male and female patients with amenable Fabry disease mutations.
  • ||||||||||  Galafold (migalastat) / Amicus
    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) -  Jan 27, 2020   
    P3,  N=76, Active, not recruiting, 
    Migalastat provided clinical benefit to patients with Fabry disease and amenable variants, regardless of disease severity. Trial completion date: Oct 2019 --> Dec 2020 | Trial primary completion date: Oct 2019 --> Dec 2020
  • ||||||||||  Galafold (migalastat) / Amicus
    Clinical, Journal:  Mutation spectrum of α-Galactosidase gene in Japanese patients with Fabry disease. (Pubmed Central) -  Dec 6, 2019   
    Additionally, 33 families (28.7%) had amenable mutations to the pharmacological chaperone migalastat. In conclusion, our study is informative when considering genetic counseling and pharmacological chaperon therapy for Fabry disease.
  • ||||||||||  Galafold (migalastat) / Amicus
    Trial completion date, Trial initiation date, Trial primary completion date:  A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease (clinicaltrials.gov) -  Dec 5, 2019   
    P3,  N=12, Not yet recruiting, 
    In conclusion, our study is informative when considering genetic counseling and pharmacological chaperon therapy for Fabry disease. Trial completion date: Jun 2020 --> Dec 2021 | Initiation date: Sep 2019 --> Mar 2021 | Trial primary completion date: Mar 2020 --> Dec 2021
  • ||||||||||  Plicera (afegostat tartrate) / Amicus
    Journal:  Assessing costs and benefits of improved soil quality management in remediation projects: A study of an urban site contaminated with PAH and metals. (Pubmed Central) -  Dec 1, 2019   
    A framework for ecological risk assessments (ERAs) within the APPLICERA - APPLICable site-specific Environmental Risk Assessment research project promotes assessments that consider other soil quality parameters than only contaminant concentrations...Although the presented Tier 3 ERA is more expensive and time-consuming than the more traditional Tier 1 ERA approach, it has the potential to lower the costs of remediation actions, decrease greenhouse gas emissions, reduce other environmental impacts, and minimise socio-economic losses. Furthermore, the remediation actions stemming from the Tier 3 ERA were predicted to exert far less negative ES effects than the actions proposed based on the results of the Tier 1 ERA.
  • ||||||||||  Galafold (migalastat) / Amicus
    Journal:  Migalastat: A Review in Fabry Disease. (Pubmed Central) -  Nov 29, 2019   
    Migalastat was generally well tolerated in both of these trials. Given its convenient oral regimen and the limited therapeutic options available, migalastat is an important treatment option for Fabry disease in patients with migalastat-amenable GLA mutations.
  • ||||||||||  Galafold (migalastat) / Amicus
    Preclinical, Journal:  Inter-assay variability influences migalastat amenability assessments among Fabry disease variants. (Pubmed Central) -  Nov 21, 2019   
    Linear regression and Bland-Altman analyses, comparing data from all variants with and without migalastat, provided additional evidence for a lack of assay reproducibility. Data from the GLP-HEK assay (and the resulting classification of amenability) can determine treatment strategy and, ultimately, patient outcomes, so discrepancies between amenability assay data could be a cause for concern for physicians managing patients with Fabry disease.
  • ||||||||||  scAVV9.CB.CLN6 / Amicus
    Enrollment closed, Gene therapy:  Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease (clinicaltrials.gov) -  Nov 2, 2019   
    P1/2,  N=13, Active, not recruiting, 
    Data from the GLP-HEK assay (and the resulting classification of amenability) can determine treatment strategy and, ultimately, patient outcomes, so discrepancies between amenability assay data could be a cause for concern for physicians managing patients with Fabry disease. Enrolling by invitation --> Active, not recruiting
  • ||||||||||  larazotide (AT1001) / Innovate Biopharmaceuticals, Galafold (migalastat) / Amicus
    Journal:  On-target action of anti-tropomyosin drugs regulates glucose metabolism. (Pubmed Central) -  Oct 10, 2019   
    Both compounds suppressed ISGU in WT muscle, but in the KO muscle there was little impact of the drugs. Collectively, this data indicates that the ATM drugs affect glucose metabolism in vivo by inhibiting Tpm3.1's function with few off-target effects.
  • ||||||||||  Galafold (migalastat) / Amicus
    Trial completion date, Trial primary completion date:  German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. (clinicaltrials.gov) -  Oct 8, 2019   
    P=N/A,  N=60, Active, not recruiting, 
    Collectively, this data indicates that the ATM drugs affect glucose metabolism in vivo by inhibiting Tpm3.1's function with few off-target effects. Trial completion date: Sep 2019 --> Jul 2020 | Trial primary completion date: May 2019 --> Jul 2020
  • ||||||||||  AT-GTX-502 / Amicus
    Trial completion date, Trial primary completion date, Gene therapy:  Gene Therapy for Children With CLN3 Batten Disease (clinicaltrials.gov) -  Oct 3, 2019   
    P1/2,  N=7, Recruiting, 
    Trial completion date: Sep 2019 --> Jul 2020 | Trial primary completion date: May 2019 --> Jul 2020 Trial completion date: Dec 2022 --> Sep 2023 | Trial primary completion date: Dec 2022 --> Jun 2023
  • ||||||||||  scAVV9.CB.CLN6 / Amicus
    Enrollment open, Trial completion date, Trial primary completion date, Gene therapy:  Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease (clinicaltrials.gov) -  Aug 28, 2019   
    P1/2,  N=13, Enrolling by invitation, 
    Trial completion date: Dec 2022 --> Sep 2023 | Trial primary completion date: Dec 2022 --> Jun 2023 Active, not recruiting --> Enrolling by invitation | Trial completion date: Mar 2023 --> Dec 2021 | Trial primary completion date: Mar 2022 --> Oct 2021
  • ||||||||||  Galafold (migalastat) / Amicus
    Enrollment open:  GALAFAB: A Study of Migalastat in Fabry Disease (clinicaltrials.gov) -  Aug 20, 2019   
    P=N/A,  N=21, Recruiting, 
    Hence, the gintonin-mediated astrocytic energy that is modulated via LPA receptors helps to protect astrocytes under hypoxia and re-oxygenation stresses. Not yet recruiting --> Recruiting