Any disease, disorder, illness, or condition affecting fewer than 200,000 people is considered rare, according to the U.S. Food and Drug Administration. There are approximately 7,000 rare diseases affecting approximately 30 million people in the U.S., while more than 90% of rare diseases have no FDA-approved treatment.

The CDISC's Therapeutic Area User Guides provide examples and guidance on implementing CDISC standards so that data can be structured effectively and easily analyzed. CDISC standards drive operational efficiencies within the organizations that use them, expedite the regulatory review process, and reduce time to market for treatments developed based on patient data.

New report investigates availability of orphan drugs
Findings of a new study commissioned by the National Organization for Rare Disorders explores the effect of patents and exclusivity on orphan drug products.

NORD announces 2021 Rare Impact Award honorees
The National Organization for Rare Disorders (NORD) announced the honorees for the 2021 Rare Impact Awards.

Global Genes produces series on gene therapy, editing
Rare disease patient advocacy organization Global Genes will publish a multimedia series about advances in gene therapy and gene editing technology throughout...

Development 'cookbooks' could speed up gene therapy
Great strides have been made in the development of gene editing tools in the past decade, but translating the technology into real therapies to treat...

Bluebird Bio to split business
Bluebird Bio announced that it will separate its severe genetic and oncology businesses into two distinct and independent publicly traded companies. Bluebird...