Editor’s Choice: Genetic Testing, Orphan Drug Designation, and The Real Age CF Starts

Happy Friday, Patient Worthians!

Today, we’re highlighting a story about the donors who sacrifice their kidneys for rare patients, an update on CF research, and an exciting orphan drug designation. We also have a story about a college student advocating for change.

Sit back and enjoy this week’s Editor’s Choice.

Transplants Can Be Essential For People With Chronic Kidney Disease. But What Are The Consequences For Donors?

Who measures the longterm health of kidney donors?

Check it out here.

The Effects of Cystic Fibrosis Begin Earlier Than Previously Thought, According To A Recent Study

When do CF symptoms begin? Possibly earlier than we realized.

Read more about it here.

College Student with Lipodystrophy Advocates for a Change in Genetic Testing

After facing rare disease challenges herself, a Rutgers student decided to become a voice for change.

Check it out here.

An Experimental Drug for Batten Disease Just Got Orphan Drug Designation From The EMA

Good news for families waiting for a Batten disease treatment! A new therapy just received a designation that will speed up the process.

Read about it here.


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

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