Tag: Rare Disease

Fibrodysplasia ossificans progressiva (FOP) remains one of the most complex and devastating ultra-rare genetic disorders known to medicine. Affecting approximately one in a million people worldwide, the condition progressively transforms soft connective tissue into bone, leading to severe mobility limitations, respiratory complications and reduced life expectancy. Recent global estimates suggest that roughly 1,000 individuals are […]

Ehlers-Danlos syndromes (EDS) represent a group of inherited connective tissue disorders characterized by joint hypermobility, tissue fragility, chronic pain and multisystem complications.  Hypermobile EDS and related hypermobility spectrum disorders are considered to affect as many as one in 500 individuals, indicating a substantially higher prevalence than once recognized.  Despite this growing awareness, delayed diagnosis, fragmented […]

The Emily Whitehead Foundation is moving toward direct patient and caregiver support while also engaging with structural payment and regulatory challenges George Eastwood has spent much of his career close to the science and manufacturing that make advanced therapies possible. Coming from the cell and gene therapy “tool space,” he worked in areas such as […]