With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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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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These Two Canadian Brothers Have Cystic Fibrosis, But Only One Has Access to Effective Treatment
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These Two Canadian Brothers Have Cystic Fibrosis, But Only One Has Access to Effective Treatment

According to a story from globalnews.ca, Andre and Joshua Laroque are nine and seven years old respectively. The brothers, being close in age, spend a lot of time together and…

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This Business Owner and Baker’s Daughters Will Both Benefit from Newly Covered Cystic Fibrosis Drugs
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This Business Owner and Baker’s Daughters Will Both Benefit from Newly Covered Cystic Fibrosis Drugs

According to a story from dailypost.co.uk, Richard Mason, the millionaire founder of moneysupermarket.com, and and baker Alison Faire's daughters Annabelle (aged 4 years) and Imogen (aged 7 years) don't have…

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Symptom Severity in Rare Disease Varies by Patients: Genetics May be to Blame

Geisinger was founded by Abigail Geisinger over 100 years ago. It now has 13 hospitals and 2 research centers. They just released a new study examining the severity of symptoms…

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A Woman With Cystic Fibrosis and Another With Heart Failure Got Organ Transplants From the Same Donor
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A Woman With Cystic Fibrosis and Another With Heart Failure Got Organ Transplants From the Same Donor

According to a story from theage.com.au, a remarkable turn of fate would bring together two women who would have otherwise never known each other---and save both of their lives in…

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Canadians with cystic fibrosis being denied access to life-changing drug, Taber man says

LETHBRIDGE — A Taber, Alta., man is calling on government to take action and help grant Canadians with cystic fibrosis better access to breakthrough treatments. Marten Devlieger was diagnosed with…

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Despite a Newly Approved Treatment, The Cystic Fibrosis Foundation Isn’t Slowing Down its Efforts
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Despite a Newly Approved Treatment, The Cystic Fibrosis Foundation Isn’t Slowing Down its Efforts

According to a story from statnews.com, the approval of the new cystic fibrosis drug Trikafta has caused quite a buzz in this rare disease community. While this new drug offers…

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ICYMI: 90% of Cystic Fibrosis Patients Could Have a New Treatment Option Thanks to This FDA Approval!

 "We went from being able to treat no patients to being able to treat nearly all patients in seven years."  90% of cystic fibrosis (CF) patients now have a new treatment…

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First Patient Dosed in Clinical Trial of Cystic Fibrosis with Severe Exocrine Pancreatic Insufficiency
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First Patient Dosed in Clinical Trial of Cystic Fibrosis with Severe Exocrine Pancreatic Insufficiency

According to a story from BioSpace, the biopharmaceutical company AzuRx BioPharma Inc. recently announced that the first patient has been treated in the company's phase 2 clinical trial. This trial…

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Man with Cystic Fibrosis Goes From 24/7 Oxygen Tank to an Avid Biker 24 Years After his Lung Transplant
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Man with Cystic Fibrosis Goes From 24/7 Oxygen Tank to an Avid Biker 24 Years After his Lung Transplant

  When Frankie Avila first got his lung transplant, he was told that, like most organs that are transplanted, his lungs only have a certain amount of time before they…

Continue Reading Man with Cystic Fibrosis Goes From 24/7 Oxygen Tank to an Avid Biker 24 Years After his Lung Transplant
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