Results 121 to 130 of about 5,730 (195)

The inflammatory path toward type 1 diabetes begins during pregnancy. [PDF]

Nat Commun
Ahrens AP, Dias R, Hyötyläinen T, Orešič M, Triplett EW, Ludvigsson J.   +5 more
europepmc   +1 more source

Synthesis of the endogenous non-reducing end heparan sulfate disaccharides for newborn screening and diagnosis of mucopolysaccharidosis types I and II. [PDF]

Carbohydr Res
Singh G, Gholap SP, Lim S, Herbst ZM, Gelb MH.   +4 more
europepmc   +1 more source

Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series. [PDF]

Commun Med (Lond)
Pillai NR, Liu N, Li X, Li X, Ahrens-Nicklas R, Adang L, Eisengart JB, Bronken G, Gupta A, Lund TC, Whitley CB, Elsea SH, Orchard PJ.   +12 more
europepmc   +1 more source

Antibody-Based Biologics for CNS Disorders. [PDF]

Antibodies (Basel)
Hui EK, Boado RJ.
europepmc   +1 more source

Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase. [PDF]

Neurotherapeutics
Jin X, Ye Q, Wu X, Su J, Song L, Fu J, Xu Q, Luo M, Liu F, Ren C, Hu M, Liu M, An Y, Li Q, Li M, Yang Y.   +15 more
europepmc   +1 more source

Diffuse Leukoencephalopathy and Ischemic Stroke in Hunter Syndrome: Interesting Case with Review of Literature. [PDF]

Ann Indian Acad Neurol
Saini AG, Anand K, Lal P, Datta S, Bhatia V, Bhadu S, Suthar R.   +6 more
europepmc   +1 more source

Advances in Therapies for Mucopolysaccharidoses. [PDF]

Curr Issues Mol Biol
Szmydtka J, Ziemian M, Banaszak R, Ciesielska M, Ładosz J, Maliszewska U, Nowakowski A, Paszek M, Suproń G, Świętochowska Z, Tkaczuk A, Wojciechowski S, Węgrzyn G.   +12 more
europepmc   +1 more source

AMCP Market Insights: The role of managed care in improving patient care in mucopolysaccharidoses. [PDF]

J Manag Care Spec Pharm
Flavin B, Haumschild R, Klein TK, McKay K, Mok T, O'Neill C, Rice M, Stephens K, Stoltz E.   +8 more
europepmc   +1 more source

Insights from the LysoNeo prospective cohort study to improve newborn screening of lysosomal diseases. [PDF]

Commun Med (Lond)
Tebani A, Guenet D, Torre S, Arion A, Héron B, Brassier A, Belmatoug N, Ausseil J, Bruel H, Lévéque C, Goldenberg A, Gruchy N, Apetrei A, Bach N, Guerrot AM, Samaan S, Ducatez F, Marret S, Bekri S.   +18 more
europepmc   +1 more source