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.

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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