by Madaline Spencer | Jul 7, 2024
Robert Califf, MD, Commissioner of the U.S. Food and Drug Administration (FDA), discusses some of the challenges that the agency faces in regards to rare diseases. Dr. Califf notes how important it is that the FDA ensures the efficacy and safety of new...
by Madaline Spencer | Jul 2, 2024
Andra Stratton, President and Co-founder of Lipodystrophy United, discusses the challenges of lipodystrophy from a patient’s perspective. Lipodystrophies are rare metabolic disorders characterized by the loss of adipose tissue. This leads to complications such...
by Madaline Spencer | Jul 1, 2024
Márcia Waddington-Cruz, MD, PhD, Federal University of Rio de Janeiro, discusses a Phase 3 trial testing eplontersen in adult patients with hereditary transthyretin polyneuropathy. Hereditary transthyretin amyloidosis (hATTR) is a rare genetic condition...
by Madaline Spencer | Jun 28, 2024
Lisa Berry, Genetic Counselor at Cincinnati Children’s Hospital, discusses results from Fabry patient survey. Fabry disease is a rare lysosomal storage disease. This disease is characterized by a deficiency of the enzyme alpha-galactosidase (alpha-GAL)....
by Madaline Spencer | Jun 27, 2024
The U.S. Food and Drug Administration (FDA) has approved VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP). CIDP is a rare neurological disorder...
