21:45 , Apr 2, 2019 |  BC Innovations  |  Distillery Therapeutics

BIN1 protein for X-linked centronuclear myopathy

DISEASE CATEGORY: Musculoskeletal INDICATION: Musculoskeletal Mouse studies suggest BIN1 could help treat X-linked centronuclear myopathies. In a mouse model of X-linked centronuclear myopathy, systemic expression of human BIN1 decreased tibialis anterior muscle atrophy and increased...
17:52 , Aug 29, 2018 |  BC Innovations  |  Distillery Therapeutics

Musculoskeletal

INDICATION: Myotonic dystrophy type 1 (DM1) Cell culture and mouse studies suggest inhibiting miR-23b and miR-218 could help treat DM1. In myoblast cells from a DM1 patient, miR-23b and miR-218 antagomirs decreased disease-associated splicing defects...
01:10 , May 13, 2017 |  BioCentury  |  Emerging Company Profile

Dialing down dynamin

Dynacure S.A.S. is developing an antisense oligonucleotide against DNM2 that could treat all three subtypes of centronuclear myopathy, two of which are not covered by industry’s pipeline. Centronuclear myopathy (CNM) is a group of rare...
07:00 , Jul 18, 2013 |  BC Innovations  |  Distillery Techniques

Technology: Disease models

This week in techniques Approach Summary Licensing status Publication and contact information Disease models Canine model for autosomal-recessive myotubular myopathy Dogs with splicing mutations in bridging integrator 1 (BIN1) could be useful as models to...
07:00 , Jun 9, 2011 |  BC Innovations  |  Distillery Therapeutics

Indication: Musculoskeletal disease

This week in therapeutics Indication Target/marker/ pathway Summary Licensing status Publication and contact information Musculoskeletal disease Muscular dystrophy Bridging integrator 1 (BIN1) Studies in patient samples and in mice suggest restoring BIN1 function could help...