News

February 24, 2021February 24, 2021

CBJF Seminar to Address NMOSD Care, COVID-19 Concerns on March 11

News

At an educational seminar next month, experts will discuss advances in the diagnosis and care of neuromyelitis optica spectrum disorder (NMOSD), as well as the ongoing COVID-19 pandemic and ... Read more

February 22, 2021February 22, 2021

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

News, syndicated

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 ... Read more

February 17, 2021February 17, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

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, ... Read more

February 17, 2021February 17, 2021

Horizon Expands R&D Portfolio by Buying Viela Bio

News

Horizon Therapeutics is growing its rare disease portfolio by acquiring the biotechnology company Viela Bio, including the approved medicine Uplizna for treating neuromyelitis optica spectrum disorder (NMOSD). “This acquisition ... Read more

February 10, 2021February 11, 2021

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

News, syndicated

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 ... Read more

February 3, 2021February 11, 2021

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

News

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket ... Read more

January 27, 2021January 27, 2021

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

News, syndicated

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including ... Read more

January 21, 2021January 21, 2021

NORD’s Caregiver Respite Program Continues Through Pandemic

News, syndicated

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization ... Read more

January 13, 2021January 13, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

News, syndicated

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 6, 2021January 6, 2021

Relapses Can Obscure Quality-of-life Ratings in Trials

News

Experiencing disease relapses after receiving a placebo in a clinical trial may change the perception of health in participants with neuromyelitis optica spectrum disorder (NMOSD) and lead to an ... Read more