Patients & Caregivers
Radioactive Somatostatin Analog Under Review by FDA (Again) to Treat Neuroendocrine Tumors
Lutathera, the 177-Lu somatostatin analog, is a new form of targeted therapy.
Afinitor Effective in Neuroendocrine Tumors; Quality of Life Scores Not Significantly Improved
In February 2016, the FDA approved Afinitor (everolimus) for treating neuroendocrine tumors (NETs) of gastrointestinal or lung origin.
Mytesi Gets Orphan Designation for Short Bowel Syndrome
Diarrhea is a common problem for patients with short bowel syndrome.
Second FDA Review of Macrilen for AGHD Treatment Monitoring Underway
A Prescription Drug User Fee Act (PDUFA) date is set for December 30, 2017.
Orphan Drug Designation Granted to Allena for Primary Hyperoxaluria Treatment
Allena announced that the FDA has granted Orphan Drug Designation to its compound, ALLN-177, for the treatment of primary hyperoxaluria.
NDA for Macrilen to Test for Growth Hormone Deficiency Resubmitted
In 2014, the FDA sent the company a Complete Response Letter. Since then, Aeterna Zentaris has completed a new Phase 3 study.
Pivotal Prader-Willi Syndrome Study with Diazoxide to Begin Late 2017
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.
TA-46 Given Orphan Designation To Treat Achondroplasia, A Form of Dwarfism
Achondroplasia patients have 1 of 2 mutations in the FGFR3 gene that leads to abnormally high levels of FGFR3 protein.
7-Year-Old Enters Triathlons to Treat His Own Rare Disease (ROHHAD)
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.
Gino Anthony Pesi: Actor and Acromegaly Survivor
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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