LGMD (Limb Girdle Muscular Dystrophy) Awareness Day is a day dedicated to raising awareness about Limb Girdle Muscular Dystrophy, which is a group of rare genetic disorders that cause progressive weakness and wasting of the muscles.
Explore our ‘Spotlights’ section, where we shine a light on the inspiring stories and invaluable contributions of individuals with LGMD, their dedicated caregivers, supporting organizations, and the clinicians who support them.
Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement
2023-12-08T00:00:00+00:00
Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement
December 14 @ 12:00 pm - 5:00 pm
This public virtual meeting will discuss approaches and opportunities for engaging patients, patient groups, rare disease or condition experts, and experts on small population studies during the drug development process for rare diseases. The meeting will focus on how to best understand patients’ experiences living with a rare disease and how to incorporate those experiences and priorities throughout the drug development process. This includes understanding patient perspectives on the burden of their condition and any existing treatment options, as well as how their current health status and risk of disease progression may impact willingness to accept risks from treatment side effects.