This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions
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This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions

  A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…

Continue Reading This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions
Ripretinib for GIST Receives Priority Review Status in China
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Ripretinib for GIST Receives Priority Review Status in China

  According to ONCLive, ripretinib, designed to treat patients with advanced gastrointestinal stroll tumors (GIST), was given Priority Review status in China by the National Medical Products Administration. This status…

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August is Stevens-Johnson Syndrome Awareness Month: Spreading Rare Disease Awareness
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August is Stevens-Johnson Syndrome Awareness Month: Spreading Rare Disease Awareness

The month of August is recognized as Stevens-Johnson Syndrome Awareness Month. The goal of this event is to elevate awareness about Stevens-Johnson syndrome among the general public and in the…

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This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions
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This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions

  A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…

Continue Reading This New Treatment for Mantle Cell Lymphoma has the Potential for Durable Remissions
Clinical Trial Results Show Rilzabrutinib as Effective Pemphigus Treatment
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Clinical Trial Results Show Rilzabrutinib as Effective Pemphigus Treatment

  Last month, at the American Academy of Dermatology (AAD) Virtual Annual Meeting, Dr. Dedee F. Murrell, MD, explained the efficacy of rilzabrutinib for treating pemphigus. According to MDedge, many…

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New Study Suggests Targeted Next Generation Sequencing Can Aid in Identification of Menkes Disease
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New Study Suggests Targeted Next Generation Sequencing Can Aid in Identification of Menkes Disease

  In a recent press release, biopharmaceutical company Fortress Biotech announced that their study regarding ATP7A targeted next generation DNA sequencing for Menkes disease was published in Molecular Genetics and Metabolism…

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New Partnership Formed to Develop Angelman Syndrome Treatment
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New Partnership Formed to Develop Angelman Syndrome Treatment

According to a story from Globe Newswire, the biopharmaceutical company Ovid Therapeutics and the University of Connecticut School of Medicine (UConn) have announced a new license agreement and research collaboration…

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You + M.E. Registry and BioBank Seeks to Build ME/CFS Resources
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You + M.E. Registry and BioBank Seeks to Build ME/CFS Resources

  In order to better understand rare conditions and diseases, researchers sometimes create registries and biobanks full of resources. Recently, the Solve ME/CFS Initiative (Solve M.E.) announced the launch of…

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KB-3061 Earns Rare Pediatric Disease Designation for Treatment of KCNQ2-EE

  In a recent press release, Knopp Biosciences announced that their drug therapy KB-3061 was granted Rare Pediatric Disease Designation to treat patients with KCNQ2 epileptic encephalopathy (KCNQ2-EE), a rare…

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COVID-19 and Myalgic Encephalomyelitis? Dr. Fauci Points Out Connections
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COVID-19 and Myalgic Encephalomyelitis? Dr. Fauci Points Out Connections

According to a story from meaction.net, Dr. Anthony Fauci was recently asked a question about what was being done to address so-called COVID-19 "long haulers," and their potential risk of…

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July 13th is GIST Awareness Day: Spreading Rare Disease Awareness
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July 13th is GIST Awareness Day: Spreading Rare Disease Awareness

Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…

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