News

March 20, 2020March 20, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News

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 nonprofit she chairs will see ... Read more

March 20, 2020March 20, 2020

Sigilon Gets $80.3M to Support Trial of Hemophilia A Candidate SIG-001

News

Some $80.3 million in financing will support the first clinical trial of Sigilon Therapeutics’ new hemophilia A cell therapy candidate, SIG-001, the company said. The trial is expected to ... Read more

March 18, 2020March 18, 2020

Novo Nordisk Pauses 3 Clinical Trials of Concizumab Amid Safety Concerns

News

Novo Nordisk has paused three clinical trials evaluating concizumab, its experimental treatment candidate for hemophilia A and B, due to safety concerns. The three Explorer studies — the Phase 2 ... Read more

March 16, 2020March 16, 2020

New Gene Therapy Shows Promise in Hemophilia A Mouse Model, Study Shows

News

Cells derived from hemophilia A patients — genetically reprogrammed to produce a functional clotting factor VIII (FVIII) that’s missing or not working in people with the blood disease — ... Read more

March 13, 2020March 13, 2020

Kovaltry Safe and Effective Prophylactic for Hemophilia A, Long-term Study Shows

News

Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension ... Read more

March 11, 2020March 11, 2020

Young Children, Parents Struggle Socially and Emotionally, Survey Finds

News

Struggles with “mood and emotions” and feelings of social isolation or inadequate social support are more pronounced in young children with hemophilia and their parents than in age-matched healthy ... Read more

March 9, 2020March 9, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 9, 2020March 9, 2020

ClotChip, Portable Way of Analyzing Blood’s Ability to Clot, Named Breakthrough Device

News

ClotChip, a portable device designed to quickly assess the ability of a person’s blood to clot, has been designated a breakthrough device by the U. S. Food and Drug Administration ... Read more

March 6, 2020March 6, 2020

In Potential $100M Deal, Medexus Acquires Ixinity for Hemophilia B

News

In an agreement expected to exceed $100 million, Medexus Pharmaceuticals has bought global commercial rights to Ixinity, an approved recombinant factor IX therapy for hemophilia B previously marketed by Aptevo ... Read more

March 4, 2020March 5, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

News

In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more

Pin It on Pinterest