top of page

Rare is NOT rare

Some rare diseases are familar:
Cystic Fibrosis, Muscular Dystrophy, Sickle Cell Anemia

1/3 children

50% of these children will not live to see their 5th birthday

Brain Scans

The RARE problem

95%

rare diseases have no FDA approved therapy or drug

1 in 10

Americans affected by rare disease

80%

rare diseases are
genetic

6 years

average wait for diagnosis

A New Era of Rare Disease

New rare diseases are discovered
everyday with Artifical Inteligence
and Machine Learning.

Genome mapping has rapidly
improved genetic testing and
expanded the market.

Big Pharma is reluctant to divert assets to small batch rare drugs.

Researchers struggle to find manufacturers.

bottom of page