by Peter Ciszewski | Jan 21, 2020
Ruth-Anne Langan Pai, collaborative research project lead for the Castleman Disease Collaborative Network (CDCN) , and a PhD student at the University of Pennsylvania explains her award-winning study that was presented at the American Society of Hematology...
by Peter Ciszewski | Jan 20, 2020
Diego Cadavid, MD of Fulcrum Therapeutics explains facioscapulohumeral muscular dystrophy, a rare disabling disease characterized by progressive skeletal muscle loss. The disease generally begins with weakness in facial muscles but as the condition progresses, ...
by Peter Ciszewski | Jan 16, 2020
Amrita Krishnan, MD, hematologist-oncologist at the City of Hope Comprehensive Cancer Center, talks about what the future holds for managing people with multiple myeloma. Multiple myeloma is a rare hematologic cancer characterized by the expansion of malignant...
by Peter Ciszewski | Jan 15, 2020
This week, numerous studies were published in peer-reviewed journals that highlight some of the innovative work that researchers around the world are doing to advance our understanding of rare conditions. Below are some of the highlights. Luspatercept in patients with...
by Peter Ciszewski | Jan 14, 2020
Claudio Santos, MD, MBA of PTC Therapeutics provides an update on the orphan drug being developed by the company, in partnership with Roche, to treat spinal muscular atrophy (SMA). The drug, risdiplam, is a survival motor neuron-2 (SMN2) splicing modifier...
