Rare Diseases By The Numbers - September 29th

RDR Staff


Number of touchdowns by Cerebral Palsy patient Lou Stamey in High School football game.


Number of drugs approved by the FDA this week to help treat Crohn’s disease. One was Stelara for adult Crohn's disease and one was the biosimilar Amjevita that includes Crohn's disease among the 5 conditions it was granted approval.


Number of daughters Kathleen Higgens has lost to cancer due to Li Fraumeni syndrome.


There are 3 types of primary progressive aphasia (PPA) – Semantic PPA, Agrammatic PPA, and Iogopenic PPA.  The progressive neurological condition results in the loss of the ability to communicate. This week, Monty Python Alum, Terry Jones, acknowledged that he suffers from PPA.


Number of rare forms of periodic fever syndromes that the FDA approved Ilaris (canakinumab) to treat. 


Number of celebrities and Rare Disease Champions of Hope we interviewed on the Blue Carpet this past weekend.


Percent increase in the stock price of Sarepta since the FDA approval of eteplirsen last week.


The year the Orphan Drug Act was passed.


Number of views of Global Genes’ video  #zebradanceoff2016.


Estimated number of rare diseases.

50 million

The dollar amount used to create the Children's Hosptial of Philadelphia (CHOP) Roberts Collaborative for Genetics and Individualized Medicine
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