Columns

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

November 6, 2019November 1, 2019

Advancing Clinical Care Recommendations for CLN1 Disease

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

A marathon is difficult even for world-class marathoners. The best of the best have rough days, days when they turn in a subpar performance, or even days when they ... Read more

October 2, 2019September 30, 2019

9 Things We Should Know About the Race to New Treatments

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

I favor running analogies — even the name of this column pays tribute to the sport. But I’m not the first patient advocate to evoke that ancient sport of ... Read more

August 28, 2019August 28, 2019

Can Patients and Families Really Drive Therapy Development?

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

The therapy development process is a marathon, not a sprint. But unlike a marathon, the road any treatment must travel from the laboratory to the marketplace is filled with ... Read more

July 17, 2019July 15, 2019

Striking a Balanced View of Clinical Trials

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

This month marks 13 years since my sister’s diagnosis of CLN1 disease, or Batten disease. That means I’ve had 4,745 days — or 113,880 hours, or 6,832,800 minutes — ... Read more

June 26, 2019June 25, 2019

3.1 Ways to Fight Your Rare Disease

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

My life changed forever 13 years ago. In the space of a few months, I started a new job, bought my first house, got married, and searched “neuronal ceroid ... Read more

May 29, 2019May 28, 2019

My Road to Big Victories for CLN1 Disease

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

With 350 million patients, rare diseases represent the largest disease community on the planet. In fact, if all rare disease patients lived in the same country, they would represent ... Read more