A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
The U.S. Food and Drug Administration (FDA) has lifted the hold it had placed on KalVista Pharmaceuticals’ Phase 2 KOMPLETE trial of KVD824 as a treatment to prevent swelling attacks in people with hereditary angioedema (HAE). KalVista said it is working closely with study investigators and clinical sites so…
The United Arab Emirates (UAE) has approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) for preventing hereditary angioedema (HAE) attacks in patients, 12 and older. “As the first targeted oral, once-daily treatment, Orladeyo provides an important new treatment option for patients and physicians,” Henrik Balle Boysen, executive vice president and chief operating…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
The prices of three approved preventive treatments — Cinryze, Haegarda, and Takhzyro (lanadelumab) — for hereditary angioedema (HAE) must be reduced by more than half for these medications to be cost-effective. Those are the conclusions of a new report published by the Institute for Clinical and Economic Review,…
Regulatory health agencies in Canada and Switzerland are reviewing BioCryst Pharmaceuticals’ applications for the approval of Orladeyo (berotralstat) in their countries for preventing hereditary angioedema (HAE) attacks in patients ages 12 and older. The applications — to Heath Canada and Swissmedic — followed Orladeyo’s recent approvals in the…
KalVista Pharmaceuticals announced plans to soon begin enrolling patients in KOMPLETE, a Phase 2 clinical trial testing its investigational oral therapy KVD824 in preventing swelling attacks in people with hereditary angioedema (HAE) at sites in Canada, Australia, and the U.K. “Over the past month we have made substantial progress in…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
Higher blood levels of molecules involved in blood vessel formation and permeability — Tie-2, Ang-2, and VE-Cadherin — along with sE-selectin, which is involved in vascular inflammation, may help to distinguish different types of angioedema, a study suggests. Notably, Tie-2 may be a biomarker of hereditary angioedema (HAE),…
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