Not logged inEpigenetic adaptation drives monocyte differentiation into microglia-like cells upon engraftment into the retina
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SaveShare Save Share Save Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension studyDeegan, Patrick B.; Goker-Alpan, Ozlem; Geberhiwot, Tarekegn; Hopkin, Robert J.; Lukina, Elena; Tylki-Szymanska, Anna; Zaher, Atef; Sensinger, Charlotte; Gaemers, Sebastiaan J. M.; Modur, Vijay; Thurberg, Beth L.; Sharma, Jyoti; Najafian, Behzad; Mauer, Michael; DasMahapatra, Pronabesh; Wilcox, William R.; Germain, Dominique P. Share Save Safety, pharmacokinetics, and pharmacodynamics of oral venglustat in Parkinson disease patients with a GBA mutation from Japan and the rest of the world: Results from part 'I of the MOVES-PD studyPeterschmitt, M. Judith; Saiki, Hidemoto; Hatano, Taku; Gasser, Thomas; Isaacson, Stuart; Kulisevsky, Jaime; Simuni, Tanya; Takahashi, Yuji; Wills, Anne-Marie; Guedes, Leonor Correia; Svenningsson, Per; Takahashi, Ryosuke; Waters, Cheryl; Katsuno, Masahisa; Ji, Allena Ji; Gaemers, Sebastiaan J. M.; Minini, Pascal; Nembo, Blandine; Sardi, Pablo; Saubadu, Stephane; Sharma, Jyoti; Fischer, Tanya Share Save Venglustat combined with imiglucerase positively affects neurological features and brain connectivity in adults with Gaucher disease type 3Schiffmann, Raphael; Cox, Timothy M.; Ida, Hiroyuki; Mengel, Eugen; Mistry, Pramod; Crawford, Nigel; Gaemers, Sebastiaan; Ji, Allena; Peterschmitt, M. Judith; Sharma, Jyoti; Zhang, Qi; Fischer, Tanya Share Save Safety, tolerability and pharmacokinetics of oral venglustat in Parkinson disease patients with a GBA mutationPeterschmitt, M. Judith; Gasser, Thomas; Isaacson, Stuart; Kulisevsky, Jaime; Mir, Pablo; Simuni, Tanya; Wills, Anne-Marie; Guedes, Leonor Correia; Svenningsson, Per; Waters, Cheryl; Ji, Allena J.; Li, Jian; Minini, Pascal; Nembo, Blandine; Saubadu, Stephane; Sharma, Jyoti; Fischer, Tanya Share Save Venglustat in adult Gaucher disease type 3: Preliminary safety, pharmacology, and exploratory efficacy from a phase 2 trial in combination with imiglucerase (LEAP)Schiffmann, Raphael; Mengel, Eugen; Crawford, Nigel; Gaemers, Sebastiaan; Hughes, Derralynn; Jovanovic, Ana; Minini, Pascal; Sharma, Jyoti; Fischer, Tanya Share Save Development and verification of a pharmacokinetic model to optimize physiologic replacement of rhIGF-1/rhIGFBP-3 in preterm infantsChung, Jou-Ku; Hallberg, Boubou; Hansen-Pupp, Ingrid; Graham, Martin A.; Fetterly, Gerald; Sharma, Jyoti; Tocoian, Adina; Kreher, Nerissa C.; Barton, Norman; Hellstroem, Ann; Ley, David Share Save Share Save Share Save Share Save Share Save Share Save 
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