MDF staff recently attended the 2017 BIO International Convention hosted by the Biotechnology Innovation Organization (BIO). BIO offers a partnering track to patient advocacy groups, enabling meetings with companies having potentially mutual interests.
Preclinical animal efficacy data used to support the scientific rational for Ionis’ phase 1/2 clinical trial in DM1 was just published.
New fly models show a DM2 phenotype at least as severe as DM1 and may provide a platform for studies of genetic modifiers and candidate therapy screening in DM.
A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.
Susannah is a 48-year-old entrepreneur with her own real estate law practice. She also has two teenagers that keep her busy at home, and she helps care for her mother. Susannah also has DM1.
A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.
MDF strives to help National Institutes of Health (NIH) grant applicants compete for research funding.
Taking as many shots on goal as possible is the only path to approval and reimbursement of safe and effective drugs for DM.