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We Applaud FDA for Two New Draft Guidances on Rare Disease Drug Development


We Applaud FDA for Two New Draft Guidances on Rare Disease Drug Development In May, the FDA outlined their new toxicology requirements for ultra-rare diseases in their draft guidance for industry, titled “Investigational Enzyme Replacement Therapy Products: Nonclinical Assessment.” The guidance potentially allows for only three months of chronic toxicology animal studies if there are no adverse findings, which is a significant improvement in policy.

About


The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases.

Currently, there are fewer than 400 approved treatments for 7000 rare diseases affecting more than 30 million Americans. read more

Events


Recommended Unified Screening Platform Roundtable, Webinar, August 26th, 2015

Annual Rare Disease Scientific Workshop, Washington DC, September 15th, 2015

Global Genes Rare Patient Advocacy Summit, Huntington Beach CA, September 24-25th, 2015

EveryLife Foundation Community Congress Meeting, Washington DC, November 4th, 2015

Rare Voice Awards Gala, Washington DC, November 4th, 2015

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Programs and Initiatives



Foundation Programs




Supported Programs