News - NORD (National Organization for Rare Disorders)

May 24, 2022

TOPIC: Uncategorized, Press Releases, Featured News, Advocacy, RDACs

Connecticut Establishes a Permanent Rare Disease Advisory Council

Posted by Valaree DonFrancesco

May 24, Hartford, CT – Yesterday, Governor Ned Lamont signed House Bill 5500 (HB 5500) into law which includes provisions to establish a permanent Rare Disease Advisory Council (RDAC) in the state of Connecticut. Connecticut established a temporary rare disease task force in 2017 that concluded its work in 2019, but the Rare Disease Advisory Council created as part of HB… Read More

Tags: advocacy, Rare Action Network, Public Policy, Connecticut, Policy, Rare Disease Advisory Council, Project RDAC

May 13, 2022

TOPIC: Patient Stories

Summerly’s Rare Journey

Posted by Julie Ostroff

I am a parent to a daughter with genetic disorder 22q11.2 deletion syndrome (commonly known as DiGeorge syndrome). She was born on July 1, 2021 and diagnosed on July 23 through a chromosomal microarray blood test. The entire month of July was a whirlwind. Her health issues began with seizures at home around nine days old, which is what… Read More

Tags: patient stories, 22q11.2 deletion syndrome, DiGeorge syndrome

May 11, 2022

TOPIC: Press Releases, Advocacy

NORD Applauds Congressional Efforts to Restore Intent of the Orphan Drug Act

Posted by Rohan Narayanan

Washington, DC — The National Organization for Rare Disorders (NORD) applauds efforts of Congressional leaders to protect the Orphan Drug Act (ODA) and ensure proper incentives are in place to continue to foster robust rare disease drug development. Last week, leaders of the House Energy and Commerce Committee included language in their bipartisan Food and Drug Amendments… Read More

Tags: Congress, Orphan Drug Act, Senate, ODA, RARE Act, Senator, Senator Bill Cassidy, Senator Tammy Baldwin, Food and Drug Amendments of 2022, HR 7667, Retaining Access and Restoring Exclusivity Act, Catalyst Pharms., Inc. v. Becerra

May 11, 2022

TOPIC: Patients & Members, Patient Stories

NORD Hosts the Largest Gathering of a Rare, Post-Transplant Cancer in History

Posted by Rohan Narayanan

Post-Transplant Lymphoproliferative Disorder (PTLD) is a rare cancer related to organ and stem cell transplants, as well as Epstein bar disease. There is no nonprofit organization dedicated to this community and the last known gathering involved less than a dozen patients and caregivers, pre-pandemic. When NORD learned of interest in this disease to share patient and caregiver stories… Read More

Tags: Cancer, FDA, drug development, research, PTLD, US Food and Drug Administration, PFDD, EL-PFDD, Partnership, Post-Transplant Lymphoproliferative Disorder, externally led Patient-Focused Drug Development

May 9, 2022

TOPIC: Press Releases, RDACs

Maine Elevates Rare Disease Voices, Establishes State Advisory Council

Posted by Rohan Narayanan

Maine becomes the 23rd state to create a council that will help address the needs of rare disease patients and their families

May 9, 2022, Augusta, ME – Today, Maine’s rare disease community celebrated the passage of Legislative Document 972 (LD 972), which officially establishes a Rare Disease Advisory Council (RDAC) in Maine…. Read More

Tags: Rare Disease Advisory Council, RDAC, Maine, ME, Representative Margaret Craven, Legislative Document 972, LD 972

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May 24, 2022

Connecticut Establishes a Permanent Rare Disease Advisory Council

Posted by Valaree DonFrancesco

May 13, 2022

Summerly’s Rare Journey

Posted by Julie Ostroff

May 11, 2022

NORD Applauds Congressional Efforts to Restore Intent of the Orphan Drug Act

Posted by Rohan Narayanan

May 11, 2022

NORD Hosts the Largest Gathering of a Rare, Post-Transplant Cancer in History

Posted by Rohan Narayanan

May 9, 2022

Maine Elevates Rare Disease Voices, Establishes State Advisory Council

Posted by Rohan Narayanan

Maine becomes the 23rd state to create a council that will help address the needs of rare disease patients and their families

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