News

May 21, 2020May 21, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s ... Read more

May 19, 2020May 19, 2020

Tegsedi Available to FAP Patients in Spain at Reduced Cost

News

Spain’s Ministry of Health has added Tegsedi (inotersen), an injection treatment for stage 1 or 2 polyneuropathy in adults with familial amyloid polyneuropathy (FAP), to the national list of prescription medicines ... Read more

May 18, 2020May 18, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

May 12, 2020May 12, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News, syndicated

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. ... Read more

May 5, 2020May 5, 2020

‘Gel’ Eye Surgery in FAP Helps, But Usually Requires Follow-up Procedures, Study Finds

News

A type of surgery that involves removal of the eye’s “gel” can improve vision in people with familial amyloid polyneuropathy (FAP), a new study suggests. However, subsequent surgeries often ... Read more

April 28, 2020April 28, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

News, syndicated

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in ... Read more

April 23, 2020April 23, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

News, syndicated

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. ... Read more

April 21, 2020April 21, 2020

FDA Places Vutrisiran on Fast Track as Potential FAP Treatment for Adults

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Alnylam Pharmaceuticals’ vutrisiran as a potential treatment of adults with familial amyloid polyneuropathy (FAP). Fast track is intended to ... Read more

April 14, 2020April 14, 2020

Sudoscan Test of Sweat Gland Function May Improve FAP Diagnosis, Study Says

News

Sudoscan, a device that evaluates sweat gland function, may help physicians improve the diagnosis of familial amyloid polyneuropathy — distinguishing these patients from those with chronic inflammatory demyelinating polyneuropathy, ... Read more

April 7, 2020April 7, 2020

Blood NfL Levels Seen as Sensitive Marker of FAP and Its Progression

News

Levels of neurofilament light chain (NfL) — a proposed biomarker of several neurological disorders — are significantly higher in people with familial amyloid polyneuropathy (FAP) who show symptoms, compared to ... Read more

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