REVIEW
|
doi:10.20944/preprints202311.0801.v1
Subject:
Medicine And Pharmacology,
Neuroscience And Neurology
Keywords:
Dysferlinopathy; limb-girdle muscular dystrophy recessive type 2 (LGMDR2); Miyoshi myopathy; distal myopathy with anterior tibial onset (DMAT); dysferlin; membrane resealing; genetic therapy; mini-dysferlin; exon skipping
Online: 13 November 2023 (10:41:28 CET)
REVIEW
|
doi:10.20944/preprints202309.1728.v1
Subject:
Medicine And Pharmacology,
Medicine And Pharmacology
Keywords:
Fibrodysplasia ossificans progressiva (FOP); heterotopic ossification; bone morphogenetic proteins (BMPs); ACVR1; antisense therapy
Online: 26 September 2023 (05:27:10 CEST)