Niemann-Pick B - RS - 5th infusion.
Descrição
![Niemann-Pick B - RS - 5th infusion.](https://media.springernature.com/full/springer-static/image/art%3A10.1038%2Fcddis.2016.16/MediaObjects/41419_2016_Article_BFcddis201616_Fig1_HTML.jpg)
Necroptosis in Niemann–Pick disease, type C1: a potential therapeutic target
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Frontiers Expert opinion on patient journey, diagnosis and clinical monitoring in acid sphingomyelinase deficiency in Turkey: a pediatric metabolic disease specialist's perspective
Niemann-Pick B - RS - 5th infusion.
![Niemann-Pick B - RS - 5th infusion.](https://www.frontiersin.org/files/Articles/530320/fmolb-07-00057-HTML/image_m/fmolb-07-00057-g001.jpg)
Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
![Niemann-Pick B - RS - 5th infusion.](https://i1.rgstatic.net/publication/332562945_Niemann-Pick_Disease_An_Underdiagnosed_Lysosomal_Storage_Disorder/links/6182873beef53e51e122cd7e/largepreview.png)
(PDF) Niemann-Pick Disease: An Underdiagnosed Lysosomal Storage Disorder
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Investigational New Drug (IND) - The Addi and Cassi Fund
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PDF) Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial
![Niemann-Pick B - RS - 5th infusion.](https://checkrare.com/wp-content/uploads/2018/05/Niemann-Pick-Type-C.png)
Niemann-Pick Disease
![Niemann-Pick B - RS - 5th infusion.](http://www.inpda.org/wp-content/uploads/2017/10/19out2017-em-janeiro-deste-ano-a-pequena-isadora-foi-selecionada-para-participar-dos-testes-1508444984632_615x300.jpg)
New Treatment of Disease that Swells Organs Gives New Hope to 4-year-old Girl - International Niemann-Pick Disease Alliance (INPDA)
![Niemann-Pick B - RS - 5th infusion.](https://www.researchgate.net/publication/274396825/figure/fig2/AS:280128157437958@1443799165817/Time-course-of-plasma-ceramide-concentrations-following-infusion-of-olipudase-alfa-The_Q320.jpg)
PDF) Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)
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WO2019130230A1 - Heterocyclic amides as kinase inhibitors - Google Patents
![Niemann-Pick B - RS - 5th infusion.](https://static.hindawi.com/articles/crit/volume-2019/9431751/figures/9431751.fig.002.jpg)
Pulmonary Type B Niemann-Pick Disease Successfully Treated with Lung Transplantation
![Niemann-Pick B - RS - 5th infusion.](https://i1.rgstatic.net/publication/348538868_Changes_in_PCSK_9_and_Apolipoprotein_B100_in_Niemann-Pick_Disease_After_Enzyme_Replacement_Therapy_with_Olipudase_Alfa/links/60029c4b299bf140889b87c3/largepreview.png)
PDF) Changes in PCSK 9 and Apolipoprotein B100 in Niemann-Pick Disease After Enzyme Replacement Therapy with Olipudase Alfa
![Niemann-Pick B - RS - 5th infusion.](https://ars.els-cdn.com/content/image/1-s2.0-S2214426923000344-gr1.jpg)
Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II
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Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann‐ Pick Disease Type C Using Crosslinked 2‐Hydroxypropyl‐β‐cyclodextrin - Carradori - 2020 - Small - Wiley Online Library
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