ICYMI: Study Suggests Female Cancer Patients Have Better Outcomes After Treatment, Experience Worse Side Effects
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ICYMI: Study Suggests Female Cancer Patients Have Better Outcomes After Treatment, Experience Worse Side Effects

According to a publication from EurekAlert, a recent study helmed by the Royal Marsden NHS Foundation Trust suggests that female cancer patients tend to live slightly longer following treatment than…

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Enrollment Opens for Phase 3 Study of Intravenous Immunoglobulin in Primary Immunodeficiency Patients

According to a publication from BioSpace, New Jersey-based Kedrion Biopharma recently announced the enrollment of the first participant in their CARES10 study. CARES10 is a phase 3 study of a…

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The World’s First Editing of DNA in Humans Using CRISPR to Treat Leber Congenital Amaurosis

  Allergan plc, a leading global pharmaceutical company, and its partner, Editas Medicine, Inc. have been given the green light for a clinical trial designed to treat patients with severe…

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Glioblastoma Clinical Trial: The Henry Ford Cancer Institute Enrolls the World’s First Glioblastoma Patient

  According to a recent article in CheckOrphan, glioblastoma (GBM) is considered to be among the deadliest cancers in the world. Currently, treatment options are surgery, chemotherapy, and radiation. In most cases,…

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Family is Racing Against the Clock to Raise $4 million for Their Baby Boy With Rare Genetic Disease
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Family is Racing Against the Clock to Raise $4 million for Their Baby Boy With Rare Genetic Disease

  When Amber Freed and her husband Mark resorted to an in-vitro fertilization (IVF) baby they were overjoyed when they found out they were pregnant and were welcoming two new…

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Despite New Rules, Extreme Costs Make Hospitals Balk at Offering the Latest and Greatest Therapies
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Despite New Rules, Extreme Costs Make Hospitals Balk at Offering the Latest and Greatest Therapies

According to a story from statnews.com, new, groundbreaking forms of treatment, such as gene therapy and CAR-T cell therapy, are giving patients with rare genetic disorders and advanced cancers more…

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Phase 2 Study of Experimental Eosinophilic Gastritis and Eosinophilic Gastroenteritis Drug Yields Encouraging Data
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Phase 2 Study of Experimental Eosinophilic Gastritis and Eosinophilic Gastroenteritis Drug Yields Encouraging Data

According to a press release from Allakos, the Company's experimental eosinophilic gastritis and eosinophilic gastroenteritis drug candidate AK002 has met all primary and secondary endpoints in a phase 2 study…

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Primary Immunodeficiency in Children: Early Intervention is Important
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Primary Immunodeficiency in Children: Early Intervention is Important

According to a story from The Indian Express, early treatment and diagnosis of primary immunodeficiencies makes management of this group of disease much simpler as a whole. In children, the…

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This Idiopathic Pulmonary Fibrosis Clinic is Improving Patient Quality of Life
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This Idiopathic Pulmonary Fibrosis Clinic is Improving Patient Quality of Life

According to a story from Medical Xpress, the Alberta Health Services' Kaye Edmonton Interstitial Lung Disease Clinic is taking a more patient-centered approach to palliative care for patients with idiopathic…

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IWMF and PleXus Communications are Teaming up to Help Spread Waldenstrom’s Macroglobulinemia Awareness
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IWMF and PleXus Communications are Teaming up to Help Spread Waldenstrom’s Macroglobulinemia Awareness

The International Waldenstrom's Macroglobulinemia Foundation (IWMF) has recently announced an exciting new partnership that will help facilitate spreading awareness about Waldenstrom's macroglobulinemia (WM), a rare form of blood cancer. This…

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Improved Guidelines for Patient Advocacy Groups Supporting Rare Diseases and Working with Pharmaceutical Companies

A study appeared recently in the Orphanet Journal of Rare Diseases reporting that rare diseases affect 350 million people worldwide.  The definition of rare disease differs between the United States…

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An Experimental Treatment for POMC and LEPR Deficiency Obesity Appears Effective

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Rhythm Pharmaceuticals, Inc., has recently announced the results from two phase 3 clinical trials. These trials were testing the company's experimental…

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FDA Permits Retrotrope’s Phase 2/3 Study of Infantile Neuroaxonal Dystrophy Drug Candidate
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FDA Permits Retrotrope’s Phase 2/3 Study of Infantile Neuroaxonal Dystrophy Drug Candidate

According to a press release from the California-based biotechnology company Retrotrope, Inc., the Company has completed recruitment for its upcoming phase 2/3 study of RT001, an experimental drug designed for…

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