Rare Disease

When Rare Disease is a Family Affair

February 28, 2020

Watching a family member succumb to a rare and progressive disease is painful. But when that rare disease is genetic, empathy can become anxiety.

The Orphan Drug Disconnect

January 9, 2019

Decades after Congress passed the Orphan Drug Act, only about 10 percent of patients with rare diseases actually receive treatment with orphan drugs, a new report finds.  

Making Treatment Less Rare for Rare Disease Patients

February 26, 2018

Waiting is a fact of life for patients with rare diseases such as cystic fibrosis. Many of these men and women, whose disease causes wheezing and frequent lung infections, live waiting for new and effective medications.  They have to delay activities due to symptoms that make breathing a struggle.  They wait for treatment, fighting through health plan barriers such as prior authorization or awaiting the decision of opaque bureaucracies such as Medicaid pharmacy and therapeutics committees.

Rare Disease Day Spotlights Need for Research

February 28, 2017

Rare Disease Day brings international attention to the plight of patients who struggle with ALS, Gaucher disease, cystic fibrosis and other less common conditions.  This year’s theme is research, a challenge for any disease state but especially for conditions with small patient populations.

Rare Disease Day Spurs Awareness

February 24, 2015

As Rare Disease Day nears, patients, their families and the organizations that represent them are fighting for greater awareness of the conditions they face.