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February 20, 2019February 20, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

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About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The ... Read more

February 19, 2019February 19, 2019

FAP More Diverse Than Previously Believed, Review Study Contends

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A review of 542 cases of hereditary transthyretin amyloid polyneuropathy (ATTR-PN) from more than 100 countries suggests that the clinical symptoms and geographical occurrence of the disease is more ... Read more

February 12, 2019February 12, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

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Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run ... Read more

February 5, 2019February 5, 2019

Eye Issues Still Prevalent in Val30Met FAP Patients After Liver Transplant, Study Shows

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Eye problems due to transthyretin (TTR) deposition are more prevalent among patients who have had hereditary amyloidosis for longer periods of time and have undergone liver transplants, a large-scale ... Read more

February 1, 2019

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

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The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal ... Read more

January 29, 2019January 29, 2019

Vyndaqel Is Safe, Effective for People with TTR-FAP, Review Study Suggests

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Treatment with Vyndaqel (tafamidis) is a safe, non-invasive way to delay neurologic progression of transthyretin familial amyloid polyneuropathy (TTR-FAP), and preserves patients’ nutritional status and quality of life, a ... Read more

January 22, 2019January 22, 2019

Heart Problems Are Top Cause of Death for FAP Patients Who Had Liver Transplant, Study Says

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Cardiac problems are the leading cause of death among transthyretin familial amyloid polyneuropathy (ATTR, also called FAP) patients who have had a liver transplant, according to a retrospective longitudinal ... Read more

January 15, 2019January 15, 2019

Longer ATXN2 Coding Sequence Linked to Earlier TTR-FAP Onset in Val30Met Families, Study Shows

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Length of naturally occurring repeats in the sequence of the ATXN2 gene may affect the age of onset of transthyretin (TTR)-related familial amyloid polyneuropathy (FAP). The study, “Large normal alleles ... Read more

January 8, 2019January 8, 2019

Vyndaqel May Help Halt Neurological Disease Progression in Patients With Val30Met Mutation, Study Reveals

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Early diagnosis and prompt treatment with Vyndaqel (tafamidis) may help halt neurological disease progression in familial amyloid polyneuropathy (FAP) patients who are positive for the Val30Met mutation, clinical analyses of ... Read more

December 21, 2018December 21, 2018

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to ... Read more

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