News

February 18, 2021February 18, 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, rare ... Read more

February 11, 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 the ... Read more

February 4, 2021February 4, 2021

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

News, syndicated

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 costs ... Read more

January 29, 2021January 29, 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 to ... Read more

January 28, 2021January 28, 2021

LEMS Treatments Don’t Affect Calcium Channels, Study Finds

News

The molecule 3,4-diaminopyridine (3,4-DAP) — approved as Firdapse and Ruzurgi to treat people with Lambert-Eaton myasthenic syndrome (LEMS) — works to directly induce the release of chemical messengers by binding to potassium ... Read more

January 22, 2021January 22, 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 for ... Read more

January 21, 2021January 21, 2021

Cancer Found After Death of LEMS Patient with Rare Immune-mediated Disorder

News

A positive SOX1 antibody test led to a post-mortem small-cell lung carcinoma (SCLC) diagnosis in a man with paraneoplastic cerebellar degeneration (PCD) and Lambert-Eaton myasthenic syndrome (LEMS), a case study ... Read more

January 20, 2021January 20, 2021

Art Contest Salutes Creativity of Rare Disease Communities

News, syndicated

Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare Diseases to heighten awareness about rare ... Read more

January 14, 2021January 14, 2021

Rare Skin Cancer Patient’s LEMS Symptoms Ease With Antibody Treatment

News

Treatment with immunoglobulins, or antibodies — proteins that help fight off infections — successfully reduced symptoms of Lambert-Eaton myasthenic syndrome (LEMS) in a woman receiving immunotherapy for a rare type ... Read more

January 11, 2021January 11, 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 Patient ... Read more