NORD’s Advice on COVID-19: You’re Stronger Than You Think
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 ... Read more
Updates
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CHMP Favors EU Approval of Ruconest for Acute HAE Attacks in Children
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 ... Read more -
NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
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 ... Read more -
Berotralstat Lowers Rate of HAE Attacks and Possibly Their Severity, Trial Reports
Preventive treatment with berotralstat (BCX7353) significantly lowered the number of swelling attacks — including those needing treatment — and the rates of standard on-demand therapy ... Read more -
Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based ... Read more
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Study Identifies Treatments That Reduce Acute Attacks in HAE Patients With Plasminogen Mutation
People with hereditary angioedema (HAE) caused by a specific mutation in the plasminogen gene ... -
Preventive Treatment with Berotralstat Lowers HAE Attack Rate, BioCryst Says
BioCryst Pharmaceuticals presented new results from a Phase 3 clinical trial showing that preventive ... -
New Production Facility for Ruconest, Pharming’s Acute HAE Treatment, Gets FDA Nod
The U.S. Food and Drug Administration (FDA) has approved a new production facility for ... -
Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
The number of treatments for children with rare diseases has grown over the past decade, ... -
Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media ... -
Mutations May Raise Risk of Angioedema Linked to Blood Pressure Medicines
Mutations in the KCNMA1 gene may predispose certain people to angioedema caused by blood pressure ...
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Stroke Patient Develops Orolingual Angioedema After Alteplase Administration, Case Study Reports
News
Researchers from University of Chicago Medicine reported the case of a patient who developed orolingual angioedema after the administration of alteplase (sold under the brand name Activase), and suggest how physicians should proceed in case of an emergency. The study, ... Read more