Results 71 to 80 of about 795 (128)

The impact of antisense oligonucleotide (ASO) therapeutics on the future of rare disease drug discovery. [PDF]

Expert Opin Drug Discov
Ruchi R, Gupta K, Verma R, Manautou J, Zhong XB, Bahal R.   +5 more
europepmc   +1 more source

Lysosomal Ion Channels and Transporters: Recent Findings, Therapeutic Potential, and Technical Approaches. [PDF]

Bioelectricity
Kondratskyi A, Bazzone A, Rapedius M, Zerlotti R, Masson B, Sadanandan NP, Parker JL, Santinho A, Moutia M, Thiam AR, Kemp A, Seibertz F, Murciano N, Friis S, Becker N, Obergrussberger A, Barthmes M, George C, George M, Dalrymple D, Gasnier B, Newstead S, Grimm C, Fertig N.   +23 more
europepmc   +1 more source

Losartan API and Nitrosamines Impurities-Stability Profile Investigation Using HPLC, LC-MS, and In Silico Assessment. [PDF]

J Sep Sci
Martini PRR, Machioli LF, Santos BPD, Oliveira TF, Paula FR, Paim CS, Mendez ASL.   +6 more
europepmc   +1 more source

Toward an Extensible Regulatory Framework for N-of-1 to N-of-Few Personalized RNA Therapy Design. [PDF]

Ther Innov Regul Sci
Bou-Jaoudeh M, Piaton-Breda G, Pereme F, Gilbert S.   +3 more
europepmc   +1 more source

The involvement of Purkinje cells in progressive myoclonic epilepsy: Focus on neuronal ceroid lipofuscinosis.

Neurobiol Dis, 2023
Bernardi S, Gemignani F, Marchese M.
europepmc   +1 more source

Developing a scoring system for gene curation prioritization in lysosomal diseases. [PDF]

Mol Genet Metab
Vernet Machado Bressan Wilke M, Goldstein J, Groopman E, Mohan S, Waddell A, Fernandez R, Chen H, Bali D, Baudet H, Clarke L, Hung C, Mao R, Yuzyuk T, Craigen WJ, Pinto E Vairo F.   +14 more
europepmc   +1 more source

Wide-field OCT angiography for non-human primate retinal imaging. [PDF]

Biomed Opt Express
Wei X, Hormel TT, Renner L, Neuringer M, Jia Y.   +4 more
europepmc   +1 more source

Integrating N-of-1 Trials Into Learning Health Care Systems. [PDF]

JAMA Neurol
Brown JEH, Chiong W, Hutchinson N.
europepmc   +1 more source

Towards Novel Antiplasmodial Agents-Design, Synthesis and Antimalarial Activity of Second-Generation β-Carboline/Chloroquine Hybrids. [PDF]

Molecules
Penava A, Marinović M, de Carvalho LP, Held J, Piantanida I, Saftić DP, Rajić Z, Perković I.   +7 more
europepmc   +1 more source

Elevated tripeptidyl-peptidase 1 corrects multiple disease phenotypes in a mouse model of juvenile neuronal ceroid lipofuscinosis. [PDF]

Mol Ther Methods Clin Dev
Banach-Petrosky W, Larrimore KE, Sleat EH, Bazer A, Samuels B, Tan Y, Melton AC, Ichida JK, Logan TP, Lobel P, Sleat DE.   +10 more
europepmc   +1 more source