News

September 2, 2020September 2, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more

September 1, 2020September 1, 2020

Vyndaqel Remains Safe After 3 Years of Treatment, Analysis Shows

News

Vyndaqel (tafamidis meglumine) remains safe after more than three years of treatment in patients with familial amyloid polyneuropathy (FAP), with no new safety concerns raised, according to an early ... Read more

August 26, 2020August 26, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

News, syndicated

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient ... Read more

August 25, 2020August 25, 2020

Ultrasound Measurements of Nerve Size Could Help Diagnose FAP, Study Says

News

Using ultrasound to measure the size of the median nerve — which runs down the upper arm and continues into the forearm and hand — could be useful for ... Read more

August 20, 2020August 20, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. ... Read more

August 18, 2020August 18, 2020

Tegsedi Seen to Ease Neuropathy Symptoms for FAP Patients in Clinical Trial

News

Treatment with Tegsedi (inotersen) eased a range of neuropathy symptoms in people with familial amyloid polyneuropathy (FAP), new data from the NEURO-TTR Phase 2/3 trial show. The findings, “Neuropathy Symptom and ... Read more

August 10, 2020August 10, 2020

New Streaming Channel Showcases Rare Disease Films

News, syndicated

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the ... Read more

August 4, 2020August 7, 2020

Tegsedi Eases Disease Progression in FAP Patients, Trial Data Show

News

Treatment with Tegsedi (inotersen) slows neurological disease progression in people with familial amyloid polyneuropathy (FAP), results from a Phase 2/3 clinical trial show. The findings may be useful in ... Read more

July 21, 2020July 21, 2020

Long-term Onpattro Use Safely Halts FAP Progression, Phase 2 Data Show

News

Long-term treatment with Onpattro (patisiran) safely and effectively halts or reverses disease progression in people with familial amyloid polyneuropathy (FAP), final data from a two-year Phase 2 trial show. ... Read more

July 14, 2020July 14, 2020

New Nerve Imaging Tool May Aid Early FAP Diagnosis, Study Suggests

News

Magnetization transfer ratio (MTR), an MRI scanning tool based on nerve imaging, may help diagnose familial amyloid polyneuropathy (FAP) in its early stages, a study suggests. With delays in ... Read more

Pin It on Pinterest