by Madaline Spencer | Jun 17, 2024
Melody Joy Paine, founder of Imperfect JOY, discusses how she captures the lies of patients with rare diseases through photography and filmmaking. Melody tries to emphasize the person when capturing their story by focusing on what makes them who they...
by Madaline Spencer | Jun 14, 2024
Leslie Edwin, President of the Cushing’s Support and Research Foundation (CSRF), and Gretchen Jordan, Associate Director of the CSRF, discuss their personal diagnostic journeys. Cushing’s syndrome is a rare endocrine disorder caused by...
by Madaline Spencer | Jun 13, 2024
Dean Suhr, co-founder and President of the MLD Foundation, discusses the need to get metachromatic leukodystrophy (MLD) as part of the Recommended Uniform Screening Panel (RUSP) and the recently approved Lenmeldy (atidarsagene autotemcel) and its effects on families....
by Madaline Spencer | Jun 12, 2024
The U.S. Food and Drug Administration has granted accelerated approval to Iqirvo (elafibranor) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA or as monotherapy in...
by Madaline Spencer | Jun 12, 2024
Jessica Klein, Consultant for Tin Soldiers Global, discusses fibrodysplasia ossificans progressiva (FOP). FOP is a rare disorder in which skeletal muscle and connective tissue are gradually replaced by bone. This condition leads to bone formation outside...
