Atl1102 a Game Changer for DMD, page-36

GMDR2 (also known as dysferlinopathy) is a rare genetic muscle disease that is caused by mutations in the dysferlin gene that leads to significant reduction or absence of dysferlin protein levels in muscle fibers. LGMDR2 is characterized by muscle inflammation, fibrosis, adiposity (fat) and progressive weakness in the hip and shoulder area (i.e. the limb girdle) proximal muscles (those closest to the center of the body) with loss of ambulation and upper limb function in adulthood. LGMDR2 affects ~ 1 in 125,000 people3. To date, no treatments have proven to be beneficial in slowing LGMDR2 disease progression.


The above was from the announcement this Am
Prof Voit is bullish about Atl1102 re Inflammation and Fibrosis..
Bingo !! in my opinion it’s a given
$$$$$

We wait !