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January 13, 2017

TOPIC: Advocacy, Get Involved

NORD Addressing the Affordable Care Act

Posted by Christina Jensen

Many of the patients that we represent have benefited from the Affordable Care Act. While we know it has not worked for all, this law addressed some of the issues that were most challenging for patients with rare diseases, such as the ability of patients with pre-existing conditions to obtain insurance and lifetime caps on coverage.  We are preparing to… Read More

January 11, 2017

TOPIC: Advocacy, Medical, Press Releases

Orphan Drugs Represent 41 Percent of All New Medications

Posted by Jennifer Huron

Washington, D.C., January 11, 2017—Data released by the U.S. Food and Drug Administration (FDA) show that 9 of the 22 new medications approved in 2016, or 41 percent, are orphan drugs to treat rare diseases.

“The percentage of orphan drugs approved in recent years by FDA remains high and this is welcome news for the 30 million Americans with rare diseases,” said Peter L. Saltonstall,… Read More

January 9, 2017

TOPIC: Uncategorized

First Natural History Registry for Necrotizing Enterocolitis

Posted by Jennifer Huron

Naugatuck, CT, and Danbury, CT, January 09, 2017—The Morgan Leary Vaughan Fund (Morgan’s Fund) has launched the first natural history registry for Necrotizing Enterocolitis (NEC)–an inflammatory disease that leads to necrosis of the intestine. NEC currently has no cure.

“The Natural History Registry for Necrotizing Enterocolitis (NEC Registry) will provide a complete picture of each patient’s experience with NEC,” said… Read More

December 23, 2016

TOPIC: Patients & Members, Press Releases, Research

Largest-Ever Study of SYNGAP1 (MRD5), Linked to Autism, was Launched

Posted by Jennifer Huron

Research study is open to participants worldwide to advance understanding and treatments for rare disease causing non-syndromic intellectual disability ranging from mild to severe with attention deficits, impulsivity, and/or mood disorders. Seventy to eighty percent of children with SYNGAP1 also have some type of epilepsy and the SYNGAP1 gene has been linked to autism.

Houston, Texas, and Danbury, CT, December… Read More