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A Tale of Two Healthcare Systems …
What do you do when you have a terminally ill child that is very much alive, and clearly deserves...
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Charlie Gard – When Parents Are Pushed Aside
I am sure most of you are watching the sad and truly tragic story of Charlie Gard with interest....
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Calling Patients with Rare Diseases/Parents of Children with Rare Diseases
We at Putting Rare Diseases Patients First!® are calling all who fall into these categories to take our survey. We are...
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Patient Engagement – Making it More than a Buzz Word…
sThis is probably the greatest time to be alive, if you are a patient with a rare disease. Why?...
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Natural History Studies and Your Rare Disease Child
Approximately 50% of rare diseases occur in children. A rare disease may present differently in different children at different ages....
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Patients Speaking for Patients
The Webinars run by Putting Rare Diseases Patients First! are designed for patients with rare diseases, and the parents...
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Natural History Studies – Making a Contribution
On December 10th, Putting Rare Diseases Patients First! hosted its fifth Webinar. The subject was Natural History Studies. Dr. Nuria...
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Compassionate Use — The Fairness Principle
When a new drug is under development, it has to be studied in patients in well designed clinical trials. ...
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Compassionate Use – What’s in a Name?
In Romeo and Juliet, by Wiliam Shakespeare, Juliet said: “What’s in a name? That which we call a rose...
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The Compassionate Pharmaceutical Industry – Fact or Fiction?
Desmond Tutu said: “We are each made for goodness, love and compassion.” When we think of “compassion” the pharmaceutical...