Rare Disease Report

Rare Disease Report: Is Sjorgen Syndrome a Rare Disease?
By James Radke at Rare Disease Report According to the Genetic and Rare Diseases Information Center (GARD) at the National Institutes of… Continue Reading
New Data Show Survival Improvement with Adcetris in Hodgkin Lymphoma Patients
by Mathew Shanley for Rare Disease Report Earlier today, Takeda and Seattle Genetics announced promising results from its Phase 3 ECHELON-1… Continue Reading
Rare Disease Report: RASRx is the Latest Duchenne Drug To Get Orphan Status From the FDA
Thanks to Rare Disease Report for this piece. University of Southern California (USC) start-up RASRx hopes to develop a drug to treat… Continue Reading
Rare Disease Report: Learning to See Thanks to a Blind Person
Many rare disease parents gain strength and inspiration by watching their ‘disabled’ child adapt because of – and sometimes thrive in… Continue Reading
Rare Disease Report: China’s Rare Disease Policy (or Lack Thereof)
By James Radke of Rare Disease Report China is about to publish its first list of national rare diseases.   Given the population of China… Continue Reading
Rare Disease Report: 4-Year-Old Girl Contracts Rare Brain Disease After Tonsil Surgery
By Andrew Black at Rare Disease Report   She went in to have her tonsil’s removed, she came back with a rare brain disease known as acute… Continue Reading
Rare Disease Report: Making Progress in Rare Diseases
After 30 years, the Orphan Drug Act is helping small companies innovate in small patient populations. While rare diseases are, by… Continue Reading