Early Data Looks Favorable for CAR-T Cell Therapy as a Treatment For Acute Lymphocytic Leukemia
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Early Data Looks Favorable for CAR-T Cell Therapy as a Treatment For Acute Lymphocytic Leukemia

According to a story from finanznachrichten.de, the Korean biopharmaceutical company Biocure Technology, Inc., recently announced the results of a pre-clinical trial that tested CAR-T cell therapy as a treatment for acute…

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Research Reveals Relationship Between Multiple Sclerosis and Digestive System Flora
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Research Reveals Relationship Between Multiple Sclerosis and Digestive System Flora

According to a story from EurekAlert!, a recent study from the University of Zurich has illuminated the connection between the gastrointestinal flora and multiple sclerosis. The discovery illuminates the potential…

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Patient Groups and the FDA Meet For Charcot-Marie-Tooth Disease Drug Development

According to a story from Charcot-Marie-Tooth News, a recent meeting between the US Food and Drug Administration (FDA) and patient advocacy groups took place to discuss the future development of…

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This Company Just Endorsed New Patient Advocacy Guidelines
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This Company Just Endorsed New Patient Advocacy Guidelines

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme has officially endorsed a set of guidelines that outline how rare disease patient advocacy organizations and drug companies should interact…

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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development
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FDA Commissioner Releases Statement on Targeted Therapies, New Efficiency Measures, and Modernizing Development

According to a story from Pharma Voice, Dr. Scott Gottlieb, the current commissioner of the US Food and Drug Administration (FDA) has recently released a statement in regards to efforts…

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New Rare Diseases Have Been Added to the Patient Advocate Foundation’s Co-Pay Relief Program
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New Rare Diseases Have Been Added to the Patient Advocate Foundation’s Co-Pay Relief Program

The Patient Advocate Foundation has recently begun to expand the reach of its popular Co-Pay Relief program. The Co-Pay Relief program is designed to provide financial assistance to qualifying patients.…

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Experimental Treatment for WHIM Syndrome Gets Orphan Drug Designation
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Experimental Treatment for WHIM Syndrome Gets Orphan Drug Designation

According to a story from BioSpace, the biotechnology company X4 Pharmaceuticals recently announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's investigational…

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Program Aims to Help Diagnose Certain Rare Diseases More Quickly
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Program Aims to Help Diagnose Certain Rare Diseases More Quickly

According to a story from PR Newswire, a new program called The Lantern Project is working to provide a free testing program that allow doctors to more quickly diagnose patients…

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“Real World” Studies Monitor The Effects of New Pulmonary Arterial Hypertension Drugs
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“Real World” Studies Monitor The Effects of New Pulmonary Arterial Hypertension Drugs

According to a story from MD Magazine, researchers are using real-world studies, outside of the controls found in the clinical setting, to trace the impacts of multiple recently approved therapies…

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