Rare Disease Report


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).
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 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.
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.
Although there have been many successes experienced in the oncology community, more genomic data is needed.
A panel at the NORD 2018 Summit discusses how patients are the catalysts behind rare disease research and drug development.
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