The Long Sprint – a Column by Laura King Edwards

August 26, 2020August 24, 2020

Good News for CLN1 Disease and a Sprint to the Finish

Columns, The Long Sprint - a Column by Laura King Edwards

Last week, Taysha Gene Therapies announced the company has entered into license and inventory purchase agreements with Abeona Therapeutics for ABO-202, an adeno-associated virus (AAV) gene therapy for CLN1 ... Read more

August 19, 2020August 18, 2020

When Is It OK to Quit Fighting Rare Disease?

Columns, The Long Sprint - a Column by Laura King Edwards

If she were still alive, my younger sister would have celebrated her 22nd birthday today. Instead, Taylor died from CLN1 disease (Batten disease) in September 2018, more than 12 ... Read more

July 29, 2020July 24, 2020

Your Rare Disease Reading List for Quarantine: Part 2

Columns, The Long Sprint - a Column by Laura King Edwards

Part two of a series. Read part one. If you’re like much of the world, you’ve spent more time at home than usual in 2020. This is especially true ... Read more

July 22, 2020July 20, 2020

Your Rare Disease Reading List for Quarantine: Part 1

Columns, The Long Sprint - a Column by Laura King Edwards

First in a two-part series. With August around the bend, a summer reading list may seem a little last-minute. But if like much of the world, you’re still spending ... Read more

May 20, 2020May 18, 2020

Batten Disease: Life, Interrupted

Columns, The Long Sprint - a Column by Laura King Edwards

The message landed in my inbox just before lunch on Day 61 of the quarantine. I was working from home while making lunch for my 1-year-old son, juggling content ... Read more

April 22, 2020May 18, 2020

Rare Disease Therapy Development: Finding a Way Forward

Columns, The Long Sprint - a Column by Laura King Edwards

Gaining access to lifesaving or even life-altering treatments has never been easy for many rare disease patients — including those with Batten disease. But the landscape is changing. What ... Read more

March 25, 2020March 23, 2020

For Many Rare Disease Families, Self-isolation Is Nothing New

Columns, The Long Sprint - a Column by Laura King Edwards

We watched from the dock as sunlight danced on the lake’s surface — a balletic performance for a small but captive audience of one in the quiet cove. Though ... Read more

February 28, 2020February 28, 2020

My Rare Disease Day Challenge to Clinical-stage Companies

Columns, The Long Sprint - a Column by Laura King Edwards

The world will soon celebrate Rare Disease Day. The 2020 edition falls on Feb. 29 — a rare day, indeed. EURORDIS founded Rare Disease Day in 2008 to raise ... Read more

January 22, 2020January 21, 2020

Running to the Finish Line, for Taylor’s Sake

Columns, The Long Sprint - a Column by Laura King Edwards

I miss my sister. Many people in the rare disease community are surprised, even shocked, to learn that I have a full-time job — which, along with my 16-month-old ... Read more

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