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NORD RARE SUMMIT 2018
Advancing Treatment for Myelodysplastic Syndrome Through Collaboration
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).
Predicting the Rare Disease Pipeline: FDA Panel Identifies Trends & Challenges
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.
RT001 Demonstrates Arrest of Disease Progression in 2 Patients with INAD
RT001 has demonstrated arrest of disease progression in 2 patients with infantile neuroaxonal dystrophy.
FDA Panel Discusses Role of Patients in Clinical Trials for Rare Diseases
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.
How FDA's Breakthrough Therapy Designation Program Changed the Rare Disease Space
Ellen Sigal, PhD, discusses how she helped make the concept of the expedited FDA development program a reality.
Rare Tumor Patient Engagement Network: Getting Patients Involved in Cancer Research
Karlyne Reilly, PhD, discusses the Rare Tumor Patient Engagement Network and the importance of getting patients with rare conditions involved in cancer research.
Greatest Challenges Faced in Diagnosing Rare Diseases
Receiving a proper diagnosis can be one of the greatest challenges for a rare disease patient, but having the right tools can help.
Fibrolamellar Cancer Foundation President on What's Next For Rare Cancers
John Hopper, MBA, highlights advances made in the fight against rare cancers and discusses what’s next for the community.
Advances Made in Cancer Treatment, but Questions Remain
Although there have been many successes experienced in the oncology community, more genomic data is needed.
Patients: The Catalysts Behind Rare Disease Research & Drug Development
A panel at the NORD 2018 Summit discusses how patients are the catalysts behind rare disease research and drug development.
Addressing Diagnosis Challenges in the Rare Disease Space
William A Gahl, MD, PhD, discusses challenges associated with diagnosing rare diseases and how health care providers might overcome them.
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