Month: October 2021

October 29, 2021October 30, 2021

No Wasted Moments

Multiple Sclerosis News Today Columns

A friend of mine recently recommended a book to me by the multitalented Brian Doyle called “One Long River of Song.” Doyle, a devout Catholic (though I don’t think you ... Read more

October 29, 2021October 29, 2021

I’m Back in the Desert Without a Horse

COVID-19 Columns, Multiple Sclerosis News Today Columns

Well, that was fun. I’ve got my party hat on from a recent birthday (of course I have one, it’s my trusty trilby! See my avatar above) and have been ... Read more

October 29, 2021October 29, 2021

Experiences and Advice From a Student Navigating College With SMA

COVID-19 Columns, SMA News Today Columns

Life after high school leaves us with plenty of paths to choose from in our pursuit of various goals. College and careers are a few of the many options to ... Read more

October 29, 2021October 29, 2021

CAD Played Role in Acute Liver Failure of Elder Woman

Cold Agglutinin News

Cold agglutinin disease (CAD) in an elder woman worsened an underlying autoimmune condition that subsequently led to acute liver failure, a case study reports. This case highlights a need for ... Read more

October 29, 2021October 29, 2021

Grant Supports Phase 3 Trial of FCX-007 for RDEB

Epidermolysis Bullosa News

The U.S. Food and Drug Administration (FDA) has awarded Castle Creek Biosciences a research grant to support its Phase 3 clinical trial of FCX-007 (dabocemagene autoficel), an investigational gene therapy ... Read more

October 29, 2021October 29, 2021

Rare Bone Tumor Led to Cushing’s Syndrome in Child

Cushing's Disease News

A recent report described the rare case of 9-year old girl who developed Cushing’s syndrome due to a hormone-producing Ewing sarcoma, a rare type of tumor that arises in bones ... Read more

October 29, 2021October 29, 2021

CMTA Hosting 20th Patient/Family Conference Online on Nov. 6

Charcot-Marie-Tooth News, COVID-19 News

The Charcot-Marie-Tooth Association (CMTA) will hold its 20th annual Patient/Family Conference — designed to empower and connect the Charcot-Marie-Tooth community and share the latest scientific breakthroughs and resources in managing the ... Read more

October 29, 2021October 29, 2021

FDA Agrees to Phase 2 Trial of HLX-0201 in Treating Fragile X

Fragile X News Today

A Phase 2a clinical trial of HLX-0201 in treating behavioral symptoms in people with fragile X syndrome was given U.S. Food and Drug Administration (FDA) approval to open, the therapy’s developer, Healx, ... Read more

October 29, 2021October 29, 2021

NIH Study: Cost of Treating Rare Diseases ‘Large’ Relative to Other Ills

Batten Disease News

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than ... Read more

October 29, 2021November 2, 2021

NIH Study: Cost of Treating Rare Diseases ‘Large’ Relative to Other Ills

Angioedema News

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than ... Read more

October 29, 2021November 1, 2021

NIH Study: Cost of Treating Rare Diseases ‘Large’ Relative to Other Ills

Hypoparathyroidism News

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than ... Read more

October 29, 2021November 2, 2021

NIH Study: Cost of Treating Rare Diseases ‘Large’ Relative to Other Ills

FAP News Today

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than ... Read more

October 29, 2021November 2, 2021

NIH Study: Cost of Treating Rare Diseases ‘Large’ Relative to Other Ills

ANCA Vasculitis News

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than ... Read more

October 29, 2021November 1, 2021

NIH Study: Cost of Treating Rare Diseases ‘Large’ Relative to Other Ills

Ehlers-Danlos News

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than ... Read more

October 29, 2021October 29, 2021

Sometimes I Forget That I’m Still a CF Patient

Cystic Fibrosis News Today Columns

I had a routine checkup with my vascular surgeon last week. As I was pulling into the parking garage, it struck me that it had been a while since I’d ... Read more