Mary Chapman, features writer —

The Amyloidosis Foundation again is marking Amyloidosis Awareness Month, observed each March, with a “Light the Night for Amyloidosis” campaign. The initiative seeks to call attention to amyloidosis, a group of diseases that includes familial amyloid polyneuropathy (FAP), a progressive disorder caused by the accumulation of amyloid fibrils…

The newly approved familial amyloid polyneuropathy (FAP) treatment Amvuttra (vutrisiran) is being offered at UC San Diego Health to help mitigate symptoms resulting from the nerve damage that marks the progressive disorder. According to UC San Diego Health, it’s the nation’s first health system to offer the treatment to…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

To mark Amyloidosis Awareness Month, observed each March, the Amyloidosis Foundation is reprising its “Light the Night for Amyloidosis” campaign. The month-long initiative is aimed at heightening awareness about amyloidosis, a group of rare disorders that also includes familial amyloid polyneuropathy (FAP), a progressive disease caused by the…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…

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…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

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…

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…

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…