Virginia Expands Medicaid Access to 400,000 Citizens, But What Does it Mean for Rare Patients?
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Virginia Expands Medicaid Access to 400,000 Citizens, But What Does it Mean for Rare Patients?

After years of resistance from the previously dominant Republican party, Virginia lawmakers were successfully able to pass a measure expanding Medicaid to nearly 400,000 more Virginians, according to the Washington…

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A New Partnership Could Lead to Better Treatments for Sjögren-Larsson Syndrome
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A New Partnership Could Lead to Better Treatments for Sjögren-Larsson Syndrome

According to a story from Business Wire, the company CENTOGENE, which is dedicated to researching rare genetic disease, will be partnering with the biotech company Aldeyra Therapeutics, Inc. This partnership…

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Researchers Announce The Development of a Possible Treatment for Hereditary Hemorrhagic Telangiectasia
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Researchers Announce The Development of a Possible Treatment for Hereditary Hemorrhagic Telangiectasia

According to a story from news-medical.net, Mariona Graupera and Frances Viñals, along with Dr. Antoni Riera-Mestre, announced the completion of research that could lead to a new potential treatment for…

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Data Shows Radiotherapy Drug is Effective and Safe for Patients With Pheochromocytoma and Paraganglioma
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Data Shows Radiotherapy Drug is Effective and Safe for Patients With Pheochromocytoma and Paraganglioma

According to a story from news-medical.net, the results of a recent research trial demonstrate that the radiation therapy drug AZEDRA (ultratrace iobenguane I131) is an effective and safe treatment option…

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Phase 2 Trial Announced for Pulmonary Arterial Hypertension Treatment
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Phase 2 Trial Announced for Pulmonary Arterial Hypertension Treatment

According to a story from BioPortfolio, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the Phase 2 trial for its proprietary, investigational product sotatercept. The medication is in development…

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Pancreatic Cancer Patients With BRCA Mutations Could Have a New Drug on the Way
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Pancreatic Cancer Patients With BRCA Mutations Could Have a New Drug on the Way

According to a story from Worldpharmanews, a PARP inhibitor drug that is commonly used to treat ovarian cancer for patients with BRCA gene mutations could also be effective against pancreatic…

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Some People Are Resistant to PARP Inhibitors, and Researchers Have Just Discovered Why
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Some People Are Resistant to PARP Inhibitors, and Researchers Have Just Discovered Why

According to a story from Ovarian Cancer News, a recent study has allowed scientists to determine why some people appear to be resistant to poly ADP ribose polymerase (PARP) inhibitors.…

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Treatment for Type 2E Limb Girdle Muscular Dystrophy Gets Rare Pediatric Drug Designation
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Treatment for Type 2E Limb Girdle Muscular Dystrophy Gets Rare Pediatric Drug Designation

According to a story from Business Wire, the gene therapy company Myonexus Therapeutics announced that the U.S. FDA has given Rare Pediatric Disease Designation for its gene therapy candidate MYO-101.…

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New Study From The FH Foundation Will Investigate Genetic Testing of Familial Hypercholesterolemia
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New Study From The FH Foundation Will Investigate Genetic Testing of Familial Hypercholesterolemia

According to a story from Cardiovascular Business, The FH Foundation, which is a frontrunning patient advocacy and research organization for familial hypercholesterolemia, recently announced that it has begun enrollment for…

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