News

Blood vessel abnormalities in the eye’s structures are common features in patients with familial amyloid polyneuropathy (FAP) caused by the Val30Met genetic mutation, according…

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to…

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Tegsedi (inotersen) as a treatment for Stage 1…

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means…

Vyndaqel (tafamidis) failed to prevent the progressive accumulation of abnormal transthyretin protein in a patient with a rare variant of familial amyloid polineuropahy (FAP), also…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials…

Researchers identified a very rare mutation in a Libyan man who was diagnosed with late-onset familial amyloid polyneuropathy (FAP), according to a case report.

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference…

Vyndaqel (tafamidis) significantly delays the progression of familial amyloid polyneuropathy (FAP) and improves the quality of life of FAP patients, a real-world study shows.