Advocacy

These collaborations will allow us to spread awareness and help expedite scientific developments for various rare diseases.

Save the Date for the 2017 NORD Rare Diseases and Orphan Products Breakthrough Summit (October 16-17).

Tri4Jett is comprised of approximately 25 triathletes who will be working with Jett McSherry to make sure he crosses the finish line.

Sparsh Shah, the 13-year-old rapper, singer, MC, and osteogenesis imperfect patient can now add inspirational speaker to his list of accomplishments.

Alternating hemiplegia of childhood involves paroxysmal and chronic neurological symptoms. MRI, EEG, EMG, CSF analysis are often normal/ non-specific in AHC patients.

Dustin Chapman has a rare disease called achalasia but that did not stop him from singing with American Idol winner, Scott McCleery.

The current situation of escalating costs for these orphan drugs would tend to indicate that prices of some drugs may be reaching a breaking point.

Ten years ago, while attending Colorado State University as a music student, Mandy lost her hearing as a result of a connective tissue disorder.

Cole learned of his rare condition in 2015 when he developed kidney failure following a viral infection.

A new model to help diagnose and/or care for undiagnosed medical conditions was recently published in Pediatrics. The model explains the value of a team approach to finding a diagnosis.