ICYMI: Addison’s Disease: Emergency Cards Issued to Patients with Adrenal Crisis Risk
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ICYMI: Addison’s Disease: Emergency Cards Issued to Patients with Adrenal Crisis Risk

by Lauren Taylor from In The Cloud Copy Adrenal insufficiency, also called Addison’s disease, is a condition in which the body does not produce adequate amounts of certain necessary hormones.…

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Living with Graves’ Disease? Then be on the Lookout for Thyroid Eye Disease
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Living with Graves’ Disease? Then be on the Lookout for Thyroid Eye Disease

Graves' Disease Awareness Month is recognized in July of each year. As part of this year's awareness campaign, the nonprofit organization Prevent Blindness and Horizon Therapeutics collaborated to create the…

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September 21-25 is Global ITP Awareness Week: Spreading Rare Disease Awareness
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September 21-25 is Global ITP Awareness Week: Spreading Rare Disease Awareness

In 2020, September 21-25 will be recognized as Global Immune Thrombocytopenia (ITP) Awareness Week. The week will aim to help spread awareness about ITP around the world both in the…

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FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug
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FDA Accepts Priority Review NDA for Potential Niemann-Pick Disease Type C Drug

According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…

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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation
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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation

According to a story from BioSpace, Ziopharma Oncology, Inc. has recently announced that its investigational therapy Ad-RTS-hIL-12 with veledimex (also called Controlled IL-12) has been given Rare Pediatric Disease designation…

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Recently Approved Treatment for Dravet Syndrome Continues to Show Impacts in Phase 3 Trial
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Recently Approved Treatment for Dravet Syndrome Continues to Show Impacts in Phase 3 Trial

According to a story from BioSpace, the biopharmaceutical company Zogenix, Inc., has recently announced the release of results from a phase 3 clinical trial testing the company's drug fenfluramine (marketed…

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September is Pulmonary Fibrosis Awareness Month: Spreading Rare Disease Awareness
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September is Pulmonary Fibrosis Awareness Month: Spreading Rare Disease Awareness

According to a story from Newswise, September is recognized as Pulmonary Fibrosis Awareness Month. This includes idiopathic pulmonary fibrosis (IPF), or disease without an identifiable cause. In the midst of…

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September 15th is World Lymphoma Awareness Day: Spreading Blood Cancer Awareness
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September 15th is World Lymphoma Awareness Day: Spreading Blood Cancer Awareness

September 15th, 2020 is recognized as World Lymphoma Awareness Day. This day is dedicated to spreading awareness about lymphoma and falls in the context of Blood Cancer Awareness Month, which…

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September 13-19 is Mitochondrial Disease Awareness Week: Spreading Rare Disease Awareness
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September 13-19 is Mitochondrial Disease Awareness Week: Spreading Rare Disease Awareness

The third week of September is recognized annually as Mitochondrial Disease Awareness Week. This global event is held in order to increase awareness of mitochondrial disease in the medical community…

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September 13-19 is Balance Awareness Week: Spreading Vestibular Disorders Awareness
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September 13-19 is Balance Awareness Week: Spreading Vestibular Disorders Awareness

This year, September 13-19 is being recognized as Balance Awareness Week. Organized by the Vestibular Disorders Association, the goal of this campaign is to help spread awareness among both the…

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COVID-19 and Heart Damage: What You Need to Know
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COVID-19 and Heart Damage: What You Need to Know

According to a story from Buzzfeed News, the coronavirus/COVID-19 pandemic continues to present new surprises and challenges to the medicine and science communities. In a recent interview, Wayne Sebastianelli, who…

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An Experimental Diagnostic for Neuroblastoma has Earned Rare Pediatric Disease Designation
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An Experimental Diagnostic for Neuroblastoma has Earned Rare Pediatric Disease Designation

According to a story from BioSpace, the radiopharmaceutical company Clarity Pharmaceuticals, Inc. has announced recently that its experimental diagnostic 64Cu-SARTATE™ has earned Rare Pediatric Disease designation from the US Food…

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New Investigative Therapy for Hereditary Angioedema Found Efficacious in Study
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New Investigative Therapy for Hereditary Angioedema Found Efficacious in Study

Ionis Pharmaceuticals has just released their findings from a study investigating the effect of IONIS-PKK-LRx and IONIS-PKKRx in hereditary angioedema (HAE) patients with different forms of the disease. These therapies work to reduce…

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Study Finds New Therapy to be Efficacious for Rheumatoid Arthritis Patients Unresponsive to Methotrexate

A new study published in the journal Rheumatology and Therapy examined the effect of baricitinib on Rheumatoid Arthritis (RA) patients who have had an inadequate to their methotrexate therapy. Methotrexate and Baricitinib…

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