Maximizing treatment efficacy through patient stratification in neuropathic pain trials

R Baron, AH Dickenson, M Calvo, SD Dib-Hajj… - Nature Reviews …, 2023 - nature.com
Abstract Treatment of neuropathic pain remains inadequate despite the elucidation of
multiple pathophysiological mechanisms and the development of promising therapeutic …

Fabry disease: Mechanism and therapeutics strategies

X Li, X Ren, Y Zhang, L Ding, M Huo, Q Li - Frontiers in pharmacology, 2022 - frontiersin.org
Fabry disease is a monogenic disease characterized by a deficiency or loss of the α-
galactosidase A (GLA). The resulting impairment in lysosomal GLA enzymatic activity leads …

Fabry disease

A Mehta, DA Hughes - Seattle (WA), 1993 - Wiley Online Library
Fabry disease is generally considered to be the second most prevalent lysosomal storage
disorder, after Gaucher disease, with an estimated incidence ranging between 1: 40,000 …

Newborn screening for Fabry disease in Northeastern Italy: results of five years of experience

V Gragnaniello, AP Burlina, G Polo, A Giuliani… - Biomolecules, 2021 - mdpi.com
Fabry disease (FD) is a progressive multisystemic lysosomal storage disease. Early
diagnosis by newborn screening (NBS) may allow for timely treatment, thus preventing …

Anderson-Fabry disease cardiomyopathy: an update on epidemiology, diagnostic approach, management and monitoring strategies

T Averbuch, JA White, NM Fine - Frontiers in Cardiovascular Medicine, 2023 - frontiersin.org
Anderson-Fabry disease (AFD) is an X-linked lysosomal storage disorder caused by
deficient activity of the enzyme alpha-galactosidase. While AFD is recognized as a …

Label-free and liquid state SERS detection of multi-scaled bioanalytes via light-induced pinpoint colloidal assembly

S Han, J Park, S Moon, S Eom, CM Jin, S Kim… - Biosensors and …, 2024 - Elsevier
Surface-enhanced Raman scattering (SERS) has been extensively applied to detect
complex analytes due to its ability to enhance the fingerprint signals of molecules around …

Newborn screening for fabry disease: current status of knowledge

V Gragnaniello, AP Burlina, A Commone… - International Journal of …, 2023 - mdpi.com
Fabry disease is an X-linked progressive lysosomal disorder, due to α-galactosidase A
deficiency. Patients with a classic phenotype usually present in childhood as a multisystemic …

Lipid and Transcriptional Regulation in a Parkinson's Disease Mouse Model by Intranasal Vesicular and Hexosomal Plasmalogen‐Based Nanomedicines

Y Wu, J Wang, Y Deng, B Angelov… - Advanced …, 2024 - Wiley Online Library
Plasmalogens (vinyl‐ether phospholipids) are an emergent class of lipid drugs against
various diseases involving neuro‐inflammation, oxidative stress, mitochondrial dysfunction …

Fabry Disease in women: Genetic basis, available biomarkers, and clinical manifestations

R Izhar, M Borriello, A La Russa, R Di Paola, A De… - Genes, 2023 - mdpi.com
Fabry Disease (FD) is a rare lysosomal storage disorder caused by mutations in the GLA
gene on the X chromosome, leading to a deficiency in α-galactosidase A (AGAL) enzyme …

Clinical characteristics, renal involvement, and therapeutic options of pediatric patients with Fabry disease

C Muntean, IM Starcea, C Stoica, C Banescu - Frontiers in pediatrics, 2022 - frontiersin.org
Inherited renal diseases represent 20% of the causes of end-stage renal diseases. Fabry
disease, an X-linked lysosomal storage disorder, results from α-galactosidase A deficient or …