May is Cystic Fibrosis Awareness Month: Spreading Rare Disease Awareness
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May is Cystic Fibrosis Awareness Month: Spreading Rare Disease Awareness

May is recognized annually as Cystic Fibrosis (CF) Awareness Month. Each year, this rare disease community comes together in order to help spread awareness about cystic fibrosis among the general…

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Exploring A Lung Disease So Rare There Are Only 7 Diagnoses: NEK10 Case Study
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Exploring A Lung Disease So Rare There Are Only 7 Diagnoses: NEK10 Case Study

When the 31-year-old woman showed up to Massachusetts General Hospital with a nasty respiratory infection, she was somewhat used to it. In fact, she had been plagued with respiratory problems…

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Rationing Care, Weighing Lives: How COVID-19 Equipment Shortages Can Harm Patients
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Rationing Care, Weighing Lives: How COVID-19 Equipment Shortages Can Harm Patients

According to the Daily Beast, the increasing spread of COVID-19 is causing real problems around the globe - and not just in the way that you might think. This viral…

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NTM Info & Research Creates Educational Tool for Patients with Bronchiectasis
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NTM Info & Research Creates Educational Tool for Patients with Bronchiectasis

According to an article in the Baker City Herald, a non-profit organization has launched a website designed to support and educate patients with bronchiectasis. NTM Info & Research advocates for patients…

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Arrowhead Pharmaceuticals Files for Clearance for Cystic Fibrosis Treatment Trial
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Arrowhead Pharmaceuticals Files for Clearance for Cystic Fibrosis Treatment Trial

Arrowhead Pharmaceuticals has recently submitted an application to the New Zealand Medicines and Medical Devices Safety Authority for the clearance of a clinical trial. They want to test their cystic…

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Handling Rare Diseases and COVID-19: National Organization for Rare Disorders Reminds You to Stay Strong
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Handling Rare Diseases and COVID-19: National Organization for Rare Disorders Reminds You to Stay Strong

  If you're worried or anxious because of COVID-19, you're not alone. If wondering about the intersection of rare diseases and COVID-19 only increases that anxiety, well, we're with you…

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Company Raises $80 Million to Develop Treatment for Cystic Fibrosis Linked to Rare Mutations
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Company Raises $80 Million to Develop Treatment for Cystic Fibrosis Linked to Rare Mutations

According to a story from Fierce Biotech, there have been some impressive advances in the treatment of cystic fibrosis recently. The latest milestone was in developing and approving the drug…

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Cystic Fibrosis Patients Are Living Longer Thanks to Improved Clinical Practices and Therapies
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Cystic Fibrosis Patients Are Living Longer Thanks to Improved Clinical Practices and Therapies

  My American Nurse recently carried an uplifting article showing increasingly positive changes for cystic fibrosis (CF) patients. A network of over one hundred thirty centers throughout the U.S. provide…

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Study Assesses Rare Disease Treatments for Children From 2010-2018
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Study Assesses Rare Disease Treatments for Children From 2010-2018

According to a story from Charcot-Marie-Tooth News, a new study highlights the advances that have been made in the last decade for treating rare diseases that affect children. However, the…

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The FDA is Developing, Approving, and Expediting Exciting New Therapies
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The FDA is Developing, Approving, and Expediting Exciting New Therapies

  Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…

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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit
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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit

  On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…

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Does the ‘Quality Adjusted Life Year’ Measure Leave Behind Rare Disease Patients?
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Does the ‘Quality Adjusted Life Year’ Measure Leave Behind Rare Disease Patients?

According to a story from Salon, the Quality Adjusted Life Year (QALY) is a statistical measure that is used in order to calculate the value of a certain medication. First…

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First Personalized Medicine Trials for Cystic Fibrosis are Underway
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First Personalized Medicine Trials for Cystic Fibrosis are Underway

Proteostasis Therapeutics has just announced results from their ex-vivo study of PTI CFTR modulators using organoids from patients living with cystic fibrosis (CF). These organoids are genetically identical, and have…

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As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action
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As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action

According to a story from newsroom.heart.org, one of the most substantial domestic policy achievements of the Obama administration was the passage of the Affordable Care Act (ACA). Since its passage,…

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Reimbursement Agreement for New Cystic Fibrosis Drug Awaits EMA Approval
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Reimbursement Agreement for New Cystic Fibrosis Drug Awaits EMA Approval

According to a story from the Irish Medical Times, Ireland's Health Service Executive (HSE) has recently announced the extension of a reimbursement agreement between the agency and the pharmaceutical company…

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