The FDA has just announced that they have granted Orphan Drug Designation to an investigative treatment called eplontersen. This therapy is being studied as a treatment option for transthyretin (ATTR)…
Dr. Douglas Marchuk, professor of genetics at Duke, has spent years studying the genetics of cardiovascular disease. According to a recent article in the Atlantic, his research at the University…
According to a recent article, trametinib has proven to be successful in treating a rare ovarian cancer subtype, which is a major advancement in treating those with this rare ovarian…
The International Consortium of FD/MAS invites you to its Film Screening and Community Discussion on the 24th of February at 7:30 pm EST. The event is free and is in…
According to a recent article, Revolo Biotherapeutics announced it has obtained approval from the Medicines and Healthcare products Regulatory Agency (MHRA) for two Phase 2 clinical trials to analyze its…
Incyte has withdrawn its New Drug Application (NDA) for parsaclisib, which is an investigational treatment for various types of lymphoma, including relapsed/refractory mantle cell lymphoma (MCL), follicular lymphoma, and marginal…
According to a recent article from BusinessWire, the European Commission (EC) has approved KEYTRUDA as a monotherapy for adult patients with renal cell carcinoma (RCC). Specifically, this approval is indicated…
On January 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted its first monthly webinar for 2022. In this month's program, the speakers provided an overview of the progress that…
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