Stiris Research Inc. Announces First Site Initiation 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 patient enrolled in a Phase II/III congenital myotonic dystrophy trial

“Enrolling the first patient in this long-term safety and efficacy study is an exciting milestone in the goal of treating congenital myotonic dystrophy (DM1), an illness with limited treatment options at this time. These patients have completed their participation in the pivotal study and are continuing to move this research forward. We look forward to working with the sites and Sponsor in advancing this potentially transformative drug,” said Lyndsay Caslick, Clinical Research Associate.

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).