The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
The European Commission has approved Pharming‘s Ruconest (conestat alfa) for the treatment of acute swelling episodes in young children with hereditary angioedema (HAE). This approval expands the age range for Ruconest, which previously was approved to treat adults and adolescents with HAE in the European Union…
A small molecule inhibitor being developed for hereditary angioedema (HAE) treatment, called PHA121, was as effective as Firazyr (icatibant acetate) — an approved therapy with a similar mechanism of action — at reversing bradykinin-induced changes in blood pressure in monkeys, results of a preclinical study show.
While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…
FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…
The European Medicines Agency has validated BioCryst Pharmaceuticals‘ application requesting the approval of berotralstat as a once-daily oral treatment to prevent swelling attacks in people with hereditary angioedema (HAE). The Committee for Medicinal Products for Human Use, an advisory committee for the European Union’s regulatory agency, will…
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…
The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency, has recommended Ruconest (conestat alfa) for approval as treatment for acute swelling episodes in children with hereditary angioedema (HAE). In the European Union, Pharming‘s Ruconest is approved for adults…
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD), said it will still take place at the…
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