Researchers Have Made an Important Microbiology Discovery Related to Cancer
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Researchers Have Made an Important Microbiology Discovery Related to Cancer

Scientists at the University of Georgia have identified a rare type of glycosylation (more on what that is in a second) which plays a major role in the function of…

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Editor’s Choice: Survivor Status and Orphan Status are Both Good Signs in the Rare Disease World

Happy Friday! This week, we have a story that might break your heart about a woman who battled cystic fibrosis. But we also have a story that might inspire you about a…

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2017 is Proving a Big Year for Orphan Drug Designation: Here’s Another One!
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2017 is Proving a Big Year for Orphan Drug Designation: Here’s Another One!

In recent news in the oncology community, the FDA has approved orphan drug designation to a new drug, SurVaxM, for glioblastoma. Glioblastoma is a rare type of brain cancer that…

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Landmark Clinical Trial Suggests the Possibility of Eradicating Multiple Myeloma
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Landmark Clinical Trial Suggests the Possibility of Eradicating Multiple Myeloma

While multiple myeloma treatment has made significant advances in recent years, the prospect of a cure remains as elusive as ever—just as it does for all cancers. But that may…

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Editor’s Choice: Soo Many Promising Results, Advancements and Treatment Options for the Rare Disease World!

TGIF Patient Worthians! This week we have promising results on promising results. First up is news on a Dravet syndrome treatment. Second is a Lupus Nephritis drug in the works! Third…

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For Multiple Myeloma Patients: Two Therapies are Better than One
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For Multiple Myeloma Patients: Two Therapies are Better than One

Results from the Phase 3 CASTOR trial from Janssen Pharmaceutical Companies were recently released, finding that Darzalex (daratumumab) was extremely effective at reducing the risk of multiple myeloma progression or…

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Relapsed or Refractory Does Not Have to Mean Untreatable
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Relapsed or Refractory Does Not Have to Mean Untreatable

At the recent Annual American Society of Clinical Oncology (ASCO) Meeting, bluebird bio, Inc. released updated data from its Phase 1 trial of bb2121. bb2121 is a new anti-BCMA chimeric…

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