May 5, 2020
Rare diseases present unique challenges, often beginning with diagnosis. Symptoms may puzzle patients and even leave health care providers uncertain.…
May 5, 2020
It’s rare for a rare disease to have an FDA-approved treatment. Yet transthyretin amyloidosis, a complex disease caused by improper protein build-up, has three.
March 19, 2020
All patients have a story about how they began experiencing symptoms, saw their doctor and came to a diagnosis.
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.
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.
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.
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.
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.
September 30, 2014
New research suggests that rare disease patients purchasing health coverage through a state or federal exchange face an all-too-common challenge: access to so-called orphan drugs.
September 2, 2014
Maximizing biologic medicine’s potential for rare disease patients means observing needed safety precautions.