Rare Disease Report
Patients & Caregivers


TED, also known as Graves’ eye disease, is an autoimmune disorder best characterized by the appearance of buldging eyes.
The survey consisted of 12 multiple-choice questions to determine the physical, emotional, and informational challenges these patients face.
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.
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