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September 12, 2017

TOPIC: Medical, Members, Press Releases, Featured News

NORD Publishes New Rare Disease Report on Shprintzen Goldberg Syndrome (SGS)

Posted by Lisa Sencen

Washington, D.C., September 15, 2017—As part of its ongoing series to promote awareness and education regarding rare diseases, the National Organization for Rare Disorders (NORD) has published a report on Shprintzen Goldberg Syndrome (SGS). This new resource is available free online to individuals around the world.

As the primary advocacy organization in the U.S. for people who have rare diseases,… Read More

August 31, 2017

TOPIC: Featured News, Medical, Members, Research, Industry, Events

FDA Announces First Gene Therapy in U.S.

Posted by Mary Dunkle

In an action the rare disease community has been eagerly awaiting, the Food and Drug Administration (FDA) has announced the first-ever approval of a gene therapy in the U.S. The product is Kymriah, and it is approved to treat certain children and young adults who have a cancer of blood and bone marrow known as acute lymphoblastic leukemia (ALL).

Approximately… Read More

August 31, 2017

TOPIC: Featured News, Medical, Members, Get Involved

Downloadable Hurricane Harvey Emergency Relief Resources

Posted by Lisa Sencen
Hurricane Harvey has left thousands rid of their belongings and forced out of their homes. For individuals living with a rare disease, the impact of such natural disasters can be even more detrimental. Together, NORD staff worked with our Texas Rare Action Network State Ambassador, Debbie Skolaski to create the Hurricane Harvey Emergency Relief Resources packet,… Read More

August 28, 2017

TOPIC: Medical, Press Releases, Featured News

NORD Publishes New Report on Sitosterolemia

Posted by Lisa Sencen

Washington, D.C., August 28, 2017—As part of its ongoing series to promote awareness and education regarding rare diseases, the National Organization for Rare Disorders (NORD) has published a report on Sitosterolemia. This new resource is available free online to individuals around the world.

Read More

August 23, 2017

TOPIC: Members

Recap: NIH NCATS Day – Partnering with Patients for Smarter Science at the NIH

Posted by Lisa Sencen

In advance of today’s Members-Only Webinar with the Director of NIH NCATS, Dr. Christopher Austin, Swapna Kakani, a summer intern with NORD, wrote the article below highlighting her experience in June at the NIH NCATS Day – Partnering with Patients for Smarter Science. We look forward to today’s special webinar with our network of Member Organizations… Read More