Author Archives: Mary Chapman

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

International Batten Disease Awareness Day is June 9

From participating in a virtual 5K to sharing family stories and videos, supporters are gearing up for International Batten Disease Awareness Day on June 9. The goal is to heighten awareness among the general public as well as  lawmakers, public authorities, industry representatives, scientists, and health professionals. Awareness and education…

Neurogene Workshop Seeks Families Affected by CLN5

Editor’s note: This story was updated March 30, 2021 to clarify that Neurogene is hosting videoconferences workshops for families in any country. Neurogene, which is developing treatments for Batten disease and other rare neurological disorders, is inviting families affected by CLN5, a form of late infantile…

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…