Results 171 to 180 of about 9,202 (225)

Safety and Efficacy of Ambroxol Therapy in Polish Patients with Gaucher Disease. [PDF]

Life (Basel)
Lipiński P, Rokicki D, Chwiałkowska K, Ciborowski M, Godzień J, Jezela-Stanek A, Korotko U, Kwaśniewski M, Niemira M, Szymańska-Rożek P, Syczewska M, Tylki-Szymańska A.   +11 more
europepmc   +1 more source

Population Pharmacokinetic/Pharmacodynamic Modeling of Therapeutic Enzymes in Lysosomal Storage Diseases. [PDF]

Clin Pharmacokinet
Barzel I, van der Ploeg AT, Pijnappel WWMP, van der Kuy PHM, van den Hout JMP, Preijers T.   +5 more
europepmc   +1 more source

Development of a Lentiviral Vector for High-Yield Production of Synthetic and Recombinant GCase for Gaucher Disease Therapy. [PDF]

Int J Mol Sci
Coelho AC, Wiezel CEV, de Campos AC, Figueiredo LLS, Suardi GAM, de Paula Bernardes J, da Cunha Tirapelli DP, Faça VM, Abraham KJ, Carlotti-Júnior CG, Siciliano V, Weiss R, Gerson S, Fontes AM.   +13 more
europepmc   +1 more source

Targeting lysosomal pH restores mitochondrial quality control in GBA1-mutant Parkinson's disease. [PDF]

Transl Neurodegener
Sheshadri P, Costa-Besada MA, Fisher A, Kiraly S, Singh K, Kourouzidou I, Blacker TS, Zeng J, Shirihai OS, Grinstaff MW, Duchen MR.   +10 more
europepmc   +1 more source

Case Report: Clinical case analysis of gaucher disease management in a resource-limited setting: a single center experience from Kashigar, Xinjiang Uygur Autonomous Region, the Western China. [PDF]

Front Pediatr
Maimaititusvn G, Kulaixi M, Atawula A, Liu F.   +3 more
europepmc   +1 more source

APOE, Aβ42, and tau differentially impact cognitive decline in Sporadic, GBA1 and LRRK2 Parkinson's disease. [PDF]

NPJ Parkinsons Dis
Botta R, Locascio JJ, Ye R, Goodheart AE, Gomperts SN.   +4 more
europepmc   +1 more source

Cytotoxic lymphocyte effector function is unaffected in patients with Gaucher disease. [PDF]

Front Immunol
Zou J, Noori T, Walterfang M, Szer J, Trapani JA, Voskoboinik I.   +5 more
europepmc   +1 more source

Gaucher disease, state of the art and perspectives. [PDF]

J Intern Med
Camou F, Berger MG.
europepmc   +1 more source

Reply: "PGRN Manifesting as Parkinson's Disease: Counseling and Treatment Implications". [PDF]

Mov Disord Clin Pract
Antonini A, Bonato G, Carecchio M.
europepmc   +1 more source

Identification of Novel Mutations in Patients Affected by Gaucher Disease. [PDF]

Int J Mol Sci
Anania M, Giacomarra M, D'Errico A, Marano M, Tartaglione I, Spada M, Pagliardini V, De Paolis MR, Giuffrida G, Duro G, Di Chiara T, Francofonte D, Marsana EM, Colomba P, Duro G, Zizzo C.   +15 more
europepmc   +1 more source