Results 231 to 240 of about 677,727 (291)

Liver Gene Therapy in Fabry Disease Mice With Low Doses of rAAV2/8 Expressing a Codon-Optimized hGLA cDNA Results in Long-Term Disease Correction. [PDF]

open access: yesJ Inherit Metab Dis
Saxena H   +11 more
europepmc   +1 more source

Novel GLA variant in Fabry cardiomyopathy: evidence of pathogenicity and amenability to migalastat. [PDF]

open access: yesOpen Heart
Fan Y   +8 more
europepmc   +1 more source

Poster Sessions

open access: yes
HemaSphere, Volume 10, Issue S1, June 2026.
wiley   +1 more source

Publication Only

open access: yes
HemaSphere, Volume 10, Issue S1, June 2026.
wiley   +1 more source

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