A new scoring system identifies key risk factors and a novel tool for assessment of DM1 patient life expectancy.
MDF is bidding a very fond farewell to John Porter, PhD, as MDF’s Chief Science Officer, and bidding a very warm hello to Elizabeth (Lisa) Ackermann, PhD.
Speech disorders (dysarthria) in CDM and childhood-onset DM1 have long been recognized and surveillance by speech and language therapists is an important aspect of patient care. Facial weakness and myotonia, and involvement of oral cavity, palatopharyngeal and respiratory muscles, are known to contribute to speech impairment.
After a new review of the literature, the question of comorbidity of childhood DM1 and autism spectrum remains an open one.
Discovery of the holy grail for DM therapy development-- drug registration endpoints -- lies in the dogged pursuit and sharing of natural history data.