More than 175 rare diseases affect the endocrine system, and Rare Disease Report wants to keep you up-to-date. Use RDR as your primary resource in researching endocrinology within the rare disease space.
Second FDA Review of Macrilen for AGHD Treatment Monitoring Underway
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.
At Rare Disease in Washington DC, we talked with Brian Wood about the years of being unable to properly get a diagnosis of extra adrenal pheochromatocytoma.
Actor Richard Hatch, star of the 80s TV show ‘Battlestar Galactica’ has passed away following his battle with stage 4 pancreatic cancer.
OPKO Health announced their phase 3, double-blind, placebo-controlled study investigating long-acting human growth hormone product (hGH-CTP) in adults with growth hormone deficiency (GHD) did not meet its primary endpoint (change in trunk fat mass).
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