Rare Disease Report
Patients & Caregivers


Lutathera, the 177-Lu somatostatin analog, is a new form of targeted therapy.
In February 2016, the FDA approved Afinitor (everolimus) for treating neuroendocrine tumors (NETs) of gastrointestinal or lung origin.
Diarrhea is a common problem for patients with short bowel syndrome.
A Prescription Drug User Fee Act (PDUFA) date is set for December 30, 2017.
Allena announced that the FDA has granted Orphan Drug Designation to its compound, ALLN-177, for the treatment of primary hyperoxaluria.
In 2014, the FDA sent the company a Complete Response Letter. Since then, Aeterna Zentaris has completed a new Phase 3 study.
It is believed that DCCR has the potential to be a safe and effective treatment for PWS patients, and a Phase III trial will finish before the end of 2017.
Achondroplasia patients have 1 of 2 mutations in the FGFR3 gene that leads to abnormally high levels of FGFR3 protein.
7-year-old Jake Vella has a rare disease called ROHHAD that saw him gain over 20 lbs in 6 months, and now he enters triathlons to keep his weight down.
Actor Gino Anthony Pesi stated in an interview with Variety that after years of pain and suffering, he began to suspect he had a rare disease acromegaly.
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