Editor’s Choice: Clinical Trials and Gala Tickets

Happy start-of-summer everyone!

We’re kicking off the summer with a wrap up on rare disease news this week. We have an article about a systemic mastocytosis study, and a clinical trial looking for a treatment for CRPS. We also have a contribution celebrating the hurdles that rare dads face, and a chance to win ~free~ tickets to an Alagille Syndrome gala.

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

Data From a Phase I Study of a Drug to Treat Systemic Mastocytosis Has Been Released

If you’ve been waiting for latest systemic mastocytosis treatment news, Blueprint Medicines made an announcement recently on the (positive!) results from a Phase I.

Check it out here.

The First Patients Have Been Enrolled on a Phase 3 Clinical Trial Evaluating a Potential CRPS Treatment

The clinical trial for Neridronate, a CRPS treatment that already has been awarded the Fast Track designation and Breakthrough Therapy designation hit a critical milestone earlier this week.

Read about it here.

Patient Worthy is Offering Two Tickets to the First Annual Alagille Syndrome Alliance Gala

Are you a caregiver, patient, support person, doctor, or other community member affected by ALGS? Patient Worthy is giving away two free tickets to the Alagille Syndrome Alliance Gala in San Diego.

Read about it here.

Fathers of Children with Rare Diseases Face Extraordinary Challenges

PW Contributor Dee Crompton is back with a touching tribute recognizing the work and battles that rare dads face every day.

Check out her story here.


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

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