Month: January 2021

January 29, 2021January 29, 2021

How I’m Staying on Top of MS’ Many ‘Gifts’

Multiple Sclerosis News Today Columns

When I was finally diagnosed with multiple sclerosis in 2009, my first question to my neurologist was, “Will I end up in a wheelchair?” She patted this question back with ... Read more

January 29, 2021January 29, 2021

Looking Back at the Good and Bad Moments of 2020

SMA News Today Columns

Nearly a month into 2021, I think it’s safe to say we’re all glad to have left 2020 behind us. It certainly wasn’t an easy year. Its hardships likely will ... Read more

January 29, 2021January 30, 2021

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

Fragile X News Today

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, 2021February 1, 2021

Irisin Hormone Levels May Help Diagnose Muscle, Bone Weakness

Cushing's Disease News

Patients with active Cushing’s disease have lower blood levels of irisin, a hormone secreted by muscle cells, than those with controlled disease and healthy people, a study has found. Thus, ... Read more

January 29, 2021January 29, 2021

Spanish Registry Seen to Capture Disease Scope, May Guide Research

Bronchiectasis News Today

The Spanish Bronchiectasis Registry (RIBRON) accurately reflects the characteristics of people with bronchiectasis in Spain, and may provide a solid basis for studies focused on multiple aspects of this disease, ... Read more

January 29, 2021January 30, 2021

Tecfidera Safe and Effective Over Long Term in Children With RRMS, Trial Shows

Multiple Sclerosis News Today

Long-term treatment with Tecfidera (dimethyl fumarate) safely and effectively reduces the frequency of relapses in children with relapsing-remitting multiple sclerosis (RRMS), according to 2.5 years of data from the FOCUS Phase 2 ... Read more

January 29, 2021January 29, 2021

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

COVID-19 News, Ehlers-Danlos News

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 29, 2021March 3, 2021

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

COVID-19 News, Lambert-Eaton News

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 29, 2021January 29, 2021

Acute Hepatic Porphyria Patients Asked to Take Part in Study Survey

Porphyria News

The American Porphyria Foundation is joining with others in asking people with acute hepatic porphyria to participate in the Porphyria Worldwide Patient Experience Research (POWER) study. Participation is in the form ... Read more

January 29, 2021January 29, 2021

FDA Issues Guidance on Developing Rare Disease Treatments

Batten Disease News

New guidelines from the U.S. Food and Drug Administration (FDA) may make it easier to develop therapies to treat extremely rare diseases, such as Batten disease. “The release of this ... Read more

January 29, 2021January 30, 2021

Rituximab Effects Evident at Low Dose, Persist After Treatment Stops

Multiple Sclerosis News Today

Rituximab shows long-term benefits, even among people with relapsing-remitting multiple sclerosis (RRMS) and clinically isolated syndrome (CIS) who stopped its use or reduced their dose, a study reported. Researchers found ... Read more

January 29, 2021January 29, 2021

Mansplaining Happens to Endometriosis Patients, Too

Endometriosis News Columns

As an endometriosis patient, I am quite versed in the art of explaining my symptoms to friends and strangers. Talking about my disease on podcasts, or telling doctors about the ... Read more

January 29, 2021January 29, 2021

High AChR Autoantibodies Linked to MG Risk After Thymectomy

Myasthenia Gravis News

High levels of autoantibodies against acetylcholine receptors (AChR) in the blood of people with thymoma — a tumor of the thymus — can help to identify patients at risk of developing myasthenia gravis ... Read more

January 29, 2021January 29, 2021

I’ve Found Powerful Camaraderie in the CF Community

Cystic Fibrosis News Today Columns

Two of the most powerful words in our vocabulary are “me, too.” Many of us tend to isolate ourselves when our circumstances are challenging. We turn inward rather than outward ... Read more

January 29, 2021January 29, 2021

How to Prepare for a Loved One’s Care as They Await Transplant

COVID-19 Columns, Pulmonary Fibrosis News Columns

My journey to becoming a caregiver for my mom, Holly, was a quick one. I learned about her prognosis and treatment options while she was en route to the ICU ... Read more