Editor’s Choice: Patient Solutions, Compassionate Use and Personal Wellbeing

Happy Friday!

This week, we examine the struggle to access experimental rare therapies through compassionate use. Also, in our new Compassion Corner series, we examine why addressing patients’ personal wellbeing can make a world of difference. And, a spotlight on CAH explores why rare clinical trials are essential to uncovering potential patient solutions.


Compassion Corner: Being Human Does Not Take Much Effort; It Only Takes 56 Seconds

Patients should be addressed not just according to their symptoms but also according to their personal wellbeing.


The Struggle to Access Experimental Rare Disease Therapies Through Compassionate Use

Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment.


Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)

Without patient volunteers, groundbreaking research and potential solutions can’t move forward.


Do you have a rare disease experience of your own? Share with us here.

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