November 21, 2021
Video
In this video interview, Titilope Fasipe shares a summary of her plenary presentation on sickle cell disease from the National Organization for Rare Disease summit.
November 18, 2021
Dr. Fasipe shares about her experience at the National Organization for Rare Disease Summit 2021 as the opening plenary speaker.
October 23, 2018
Article
A team of investigators has found that collaborative efforts among several leading medical institutions have helped increase the study of new medications for myelodysplastic syndromes (MDS).
October 19, 2018
A panel of members from the US Food and Drug Administration (FDA) sat down to discuss the influential factors and projective trajectory of the rare disease pipeline.
October 18, 2018
RT001 has demonstrated arrest of disease progression in 2 patients with infantile neuroaxonal dystrophy.
Members from the FDA came together to participate in an informative session and discuss the traditional pathway and future outlook of clinical trials in rare diseases.
October 17, 2018
Ellen Sigal, PhD, discusses how she helped make the concept of the expedited FDA development program a reality.
Karlyne Reilly, PhD, discusses the Rare Tumor Patient Engagement Network and the importance of getting patients with rare conditions involved in cancer research.
Receiving a proper diagnosis can be one of the greatest challenges for a rare disease patient, but having the right tools can help.
John Hopper, MBA, highlights advances made in the fight against rare cancers and discusses what’s next for the community.
How PREPARE Data, SMART Recommendations Alter Modern Asthma Strategy
Family History of OSA Contributes to Pathogenesis of the Condition
Significant OSA Prevalence in Pediatric Patients with Pulmonary Hypertension
