The trial aims to assess the intra-cochlear injection of the gene therapy’s tolerability, efficacy, and safety in paediatric subjects with OTOF gene-mediated hearing loss. Credit: StoryTime ...

The mucopolysaccharidosis market is witnessing a surge in innovation, driven by cutting-edge gene therapies, enzyme replacement treatments, and novel drug developments. The market is poised for ...

Jan. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II ...

Background: Cardiovascular involvement is among the main features of MPS disorders and it is also a significant cause of morbidity and mortality. The range of manifestations includes cardiac valve ...

Lymphocyte subset enumeration was performed by flowcytometry. 50µl of whole blood were mixed with anti-CD16 (clone 3G8, BD Pharmingen), -CD45-PerCP (clone 2D1, BD Biosciences), -CD19-PE-Cy7 (clone ...