Company Raises $80 Million to Develop Treatment for Cystic Fibrosis Linked to Rare Mutations
source: pixabay.com

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…

Continue Reading Company Raises $80 Million to Develop Treatment for Cystic Fibrosis Linked to Rare Mutations
Cystic Fibrosis Patients Are Living Longer Thanks to Improved Clinical Practices and Therapies
source: pixabay.com

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…

Continue Reading Cystic Fibrosis Patients Are Living Longer Thanks to Improved Clinical Practices and Therapies
The FDA is Developing, Approving, and Expediting Exciting New Therapies
source: pixabay.com

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…

Continue Reading The FDA is Developing, Approving, and Expediting Exciting New Therapies
An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit
source: pixabay.com

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…

Continue Reading An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit
Does the ‘Quality Adjusted Life Year’ Measure Leave Behind Rare Disease Patients?
source: pixabay.com

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…

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

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…

Continue Reading With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action
source: pixabay.com

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,…

Continue Reading As the ACA Faces Another Court Challenge, Patient Groups Urge Quick Action
This Business Owner and Baker’s Daughters Will Both Benefit from Newly Covered Cystic Fibrosis Drugs
source: pixabay.com

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…

Continue Reading This Business Owner and Baker’s Daughters Will Both Benefit from Newly Covered Cystic Fibrosis Drugs
A Woman With Cystic Fibrosis and Another With Heart Failure Got Organ Transplants From the Same Donor
Free-Photos / Pixabay

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…

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