Results 51 to 60 of about 925 (73)

Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I. [PDF]

open access: yesMol Ther, 2023
Cataldi MP   +8 more
europepmc   +1 more source

mRNA Degradation as a Therapeutic Solution for Mucopolysaccharidosis Type IIIC: Use of Antisense Oligonucleotides to Promote Downregulation of Heparan Sulfate Synthesis. [PDF]

open access: yesInt J Mol Sci
Santos JI   +11 more
europepmc   +1 more source

Nucleoside Phosphorylases make N7-xanthosine. [PDF]

open access: yesNat Commun
Westarp S   +9 more
europepmc   +1 more source

Prospective observational study of FKRP-related limb-girdle muscular dystrophy R9: A GRASP consortium study. [PDF]

open access: yesAnn Clin Transl Neurol
Alfano LN   +31 more
europepmc   +1 more source

NAD+ metabolism restriction boosts high-dose melphalan efficacy in patients with multiple myeloma. [PDF]

open access: yesBlood Adv
Soncini D   +27 more
europepmc   +1 more source

Structural insights into terminal arabinosylation of mycobacterial cell wall arabinan. [PDF]

open access: yesNat Commun
Liu Y   +19 more
europepmc   +1 more source

Genetic blueprint of congenital muscular dystrophies with brain malformations in Egypt: A report of 11 families. [PDF]

open access: yesNeurogenetics
Safwat S   +5 more
europepmc   +1 more source

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