FAP News Today

April 12, 2021April 12, 2021

Weight Loss, Other GI Symptoms, Common in FAP, Italian Study Finds

FAP News Today

Gastrointestinal (GI) symptoms, particularly unintentional weight loss, occur frequently in people with hereditary transthyretin amyloidosis, also known as familial amyloid polyneuropathy (FAP), according to data from an Italian study. Indeed, ... Read more

April 5, 2021April 5, 2021

European Commission Grants NTLA-2001 Gene-editing Therapy Orphan Drug Status

FAP News Today

The European Commission (EC) has granted orphan drug status to Intellia Therapeutics’ NTLA-2001 gene-editing therapy for transthyretin amyloidosis (ATTR), a group of disorders that also encompasses familial amyloid polyneuropathy (FAP). ... Read more

March 31, 2021March 31, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

FAP News Today

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of ... Read more

March 29, 2021March 29, 2021

FAP Nerve Function Found to Deteriorate Faster Than Other Disorders

FAP News Today

Nerve function deteriorates more rapidly in people with familial amyloid polyneuropathy (FAP) than in those with other peripheral nerve disorders, a literature review has found.  These findings may improve the ... Read more

March 22, 2021March 22, 2021

Scientist Awarded $1.79M to Develop New Amyloidosis Treatment

FAP News Today

Scientist Jonathan Wall, PhD, has won a $1.79-million grant to develop a potential treatment for amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The grant, from the ... Read more

March 15, 2021March 15, 2021

New Grant Program to Award ‘Young Investigators’ of ATTR Amyloidosis

FAP News Today

Ionis Pharmaceuticals has launched a new grant program to fund novice researchers investigating transthyretin (ATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP), also known as hereditary ... Read more

March 8, 2021March 8, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

COVID-19 News, FAP News Today

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 8, 2021March 8, 2021

Interim Trial Data of Gene-editing Therapy NTLA-2001 Expected This Year

FAP News Today

Intellia Therapeutics anticipates reporting interim data this year from a Phase 1 study evaluating the safety and activity profile of NTLA-2001, its investigational gene-editing therapy to treat hereditary transthyretin amyloidosis ... Read more

March 2, 2021March 2, 2021

‘Profound’ Loss Voiced by People With FAP, hATTR Amyloidosis in Interview Study

FAP News Today

People living with hereditary transthyretin (hATTR) amyloidosis — which includes familial amyloid polyneuropathy (FAP) — report “profound” life changes due to their disease, particularly as it progresses and affects them ... Read more

February 26, 2021February 26, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

COVID-19 News, FAP News Today

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more

February 24, 2021February 24, 2021

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

COVID-19 News, FAP News Today

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 ... Read more

February 23, 2021February 24, 2021

Focus Groups Highlight Need for Patient Input in Diagnosis, Treatment

FAP News Today

In focus groups, transthyretin (ATTR) amyloidosis patients and families reported challenges and concerns related to the odyssey of getting a diagnosis, the effect of symptoms on quality of life, and ... Read more

February 19, 2021February 19, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

FAP News Today

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

February 16, 2021February 16, 2021

GDF-15 Protein May Be Early Biomarker of Heart Disease

FAP News Today

The levels of blood GDF-15 could detect the development and progression of heart disease in people with hereditary transthyretin (ATTRv) amyloidosis, which includes familial amyloid polyneuropathy, a new study suggests. ... Read more

February 11, 2021February 11, 2021

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

COVID-19 News, FAP News Today

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 ... Read more