Stiris Research Inc. Announces First Treatment-Naive Subject Screening in Phase II-III Congenital Myotonic Dystrophy Trial.

Stiris Research (“Stiris”) an entrepreneurial clinical trial management company focused on the management of Phase I-IIIB trials, today announces the first treatment-naïve subject screening in a Phase II/III open label congenital myotonic dystrophy trial

“I am pleased to announce that we have screened the first Treatment Naïve patient in our Open-Label Extension Congenital and Childhood Onset Myotonic Dystrophy study. The recently released protocol amendment allows for inclusion of Childhood Onset DM1 subjects in addition to Congenital DM1. This important milestone allows for these patients to have access to the investigational product who currently have limited treatment options. We are very grateful for our investigative sites who have been pivotal in ensuring swift implementation of the protocol amendment. We are looking forward to further enrollment across all of our sites!” said Madison Pearce, Project Manager.

Congenital Myotonic Dystrophy (CMD) is a rare early childhood form of myotonic dystrophy (also known as Steinert’s disease) with symptoms generally beginning to show in childhood or later in life, however symptoms of congenital myotonic dystrophy are evident from birth. CMD is generally characterized by diminished muscle tone (hypotonia), progressive muscle weakness and degeneration (atrophy). CMD only occurs when the mother already has myotonic dystrophy (although she may not be aware of this) and passes it on to her child in a more severe form. Myotonic, meaning “involving muscle stiffness” and Dystrophy, meaning “muscle wasting, and weakness” have a devastating impact on these children. CMD varies considerably in severity, symptoms and progression from child to child, but in some cases can be fatal, especially in the early weeks of life (www.musculardystrophyuk.org) and (https://rarediseases.org/rare-diseases/congenital-muscular-dystrophy).