Accelerating access to advanced therapies for children with ultra-rare diseases.
Rare diseases are defined by their very low prevalence in the population. In Europe, a disease is classified as rare when it affects fewer than 5 in 10,000 people, and as ultra-rare when it affects fewer than 1 in 50,000. Because they’re so rare, ultra-rare diseases draw little commercial interest from the biotech and pharmaceutical industries. As a result, private investment is almost nonexistent, leaving these patients in a position of particular vulnerability.
Since 2017, Fundación Columbus has worked to accelerate access to advanced therapies for children with ultra-rare diseases. We focus on diseases with a viable therapeutic path forward, partnering with patient associations, families, hospitals, researchers, companies, and institutions to improve quality of life for children around the world.
When a treatment exists, we believe no child should be denied access to it simply because it isn’t profitable enough for the market.
+300M
People living with a rare disease, spanning nearly 7,000 distinct conditions
70%
of rare diseases begin in childhood
5%
of rare diseases have an approved treatment
Since we started, we’ve helped more than 50 children around the world with ultra-rare diseases access treatment, in many cases the only one that exists anywhere for their disease. Meet the first children who made it possible.
Many parents have developed therapies for their own children, but the real challenge is making that therapy accessible to every other affected child, without the backing of a pharmaceutical company. We currently support access to therapies for AADC, SPG50, CLN7, CMT4J, and CTNNB1, all in clinical trials. Our goal is to provide access to 10 therapies for 10 diseases.
Will you help us get there?
We advance programs
for ultra-rare diseases,
cutting through the barriers
that slow down research,
manufacturing, and
clinical knowledge.
We bring together
the private sector,
researchers, patient associations,
and the medical community
to push new therapies
forward.
We connect medical teams,
institutions, and manufacturers
to deliver gene therapies
to the families and children
who need them.
Children with ultra-rare diseases treated
Advanced therapies supported
Phase 3 clinical trial in the EU
Cultural, sporting, and scientific events organized
Along the way, we’ve chosen partners who help us get where we can’t go alone.
A leading international center
for pediatric care and clinical trials,
bringing world-class expertise and
commitment to expanding options
for children with ultra-rare diseases.
A nonprofit dedicated
to identifying and developing
advanced therapies through
to clinical application, and to
opening the door to philanthropy
from the United States.
Your support gives children with ultra-rare diseases, and their families, a second chance: to be supported, heard, and carried forward by science that becomes hope.
