Hope for Patients with Early Alzheimer’s Disease and Their Family Members

Hope for Patients with Early Alzheimer’s Disease and Their Family Members By Kathleen Hoffman, PhD, MSPH Losing who you are is essentially what the diagnosis of Alzheimer’s disease means. An irreversible and progressive brain disorder, it is the sixth leading cause of death, affecting over 6 million people in the US.1 The disease profoundly impacts those who are diagnosed as well as their loved ones: I am writing today because I'm having a hard time accepting my husband's diagnosis of Alzheimer's Disease. He is only [age] and has no family history of the disease. . . They [...]

Listen to People Impacted by Rare Disease

Listen to People Impacted by Rare Disease By Kathleen D. Hoffman, PhD “The success (or failure) of the majority of rare disease drug development programmes rests on surrogate outcomes (e.g. laboratory measures, organ size) that may not reflect treatment benefits that patients value.”1 ~Morel and Canto Thomas Morel and Stefan Canto’s position paper from 2017 clearly describes the dilemma faced in rare disease research. Lab measures and organ size don’t speak to what patients with rare diseases experience, need or value. This focus on measures that are not meaningful impacts the development of rare disease treatments [...]