Supplemental New Drug Application Submitted For Potential Acute Myeloid Leukemia Treatment
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Supplemental New Drug Application Submitted For Potential Acute Myeloid Leukemia Treatment

According to a story from PR Newswire, the biopharmaceutical company AbbVie has recently announced that it has sent a supplemental New Drug Application to the FDA for its product venetoclax,…

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American Society for Clinical Oncology’s Annual Meeting: Summary of Rare Cancer Findings
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American Society for Clinical Oncology’s Annual Meeting: Summary of Rare Cancer Findings

According to a story from currentargus.com, the annual meeting of the American Society for Clinical Oncology, which takes place in Chicago, is the largest convergence dedicated to cancer research in…

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New Drug Approved in the EU for Treating Hereditary Transthyretin Amyloidosis
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New Drug Approved in the EU for Treating Hereditary Transthyretin Amyloidosis

According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…

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How Rare is Eosinophilic Esophagitis Anyway? A Family’s Story Brings Awareness
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How Rare is Eosinophilic Esophagitis Anyway? A Family’s Story Brings Awareness

According to a story from the Washington Times-Reporter, Michelle Marcinak's three sons-- John Henry, Leo, and Rafe-- were all diagnosed with the rare gastric condition eosinophilic esophagitis. Early symptoms of…

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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation
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Investigational Drug for Facioscapulohumeral Muscular Dystrophy Gets Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…

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Kids With Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis Experience Complications and Get Inconsistent Treatment, Study Says
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Kids With Stevens-Johnson Syndrome and Toxic Epidermal Necrolysis Experience Complications and Get Inconsistent Treatment, Study Says

According to a story from ScienceDaily, a recent analysis has been conducted in order to investigate patient outcomes for children with the rare skin condition Stevens-Johnson syndrome, as well its…

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Online Community Helped Free a Family Affected by Coffin-Siris Syndrome From Isolation

According to a story from the Globe and Mail, Michelle Quinlan was overwhelmed when her child Avery was diagnosed with Coffin-Siris syndrome, a rare genetic disease that can cause intellectual…

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Positive Genetic Test Results for Rare Disease Risk are not Necessarily a Cause for Alarm

According to a story from Global News, direct-to-consumer genetic tests are becoming increasingly popular. These tests can be used to learn new info about your ancient ancestors, closer relatives, and,…

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A New Treatment for Dupuytren’s Contracture Could Improve Outcomes

According to a story from the Deccan Chronicle, researchers may have discovered a new treatment for Dupuytren's contracture that could offer substantial benefits over currently available treatment options. The scientists…

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Study Offers Free Screening for Cancer and BRCA Mutations for Ashkenazi Jewish Community
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Study Offers Free Screening for Cancer and BRCA Mutations for Ashkenazi Jewish Community

According to a story from Jewish Community Voice, the BRCA Founder Outreach Study, which is a national, cancer-focused research study, is offering free screenings for people of Ashkenazi Jewish descent.…

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Scientists Have Discovered a Mechanism Behind Hypokalemic Periodic Paralysis
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Scientists Have Discovered a Mechanism Behind Hypokalemic Periodic Paralysis

According to a story from sciencedaily.com, recent research has revealed the mechanism that causes hypokalemic periodic paralysis, a rare condition that causes patients to experience sudden, severe muscle weakness. The…

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Unraveling the Mysteries of Familial Mediterranean Fever Led to a Career of Rare Disease Research
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Unraveling the Mysteries of Familial Mediterranean Fever Led to a Career of Rare Disease Research

According to a story from govloop.com, Dr. Daniel Kastner, the Scientific Director for the Division of Intramural Research at the National Institutes of Health (NIH), has committed his career to…

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