-
1 week ago | 
hemophilianewstoday.com | Margarida Maia |Jennifer Lynne |Marisa Wexler |Mary Chapman
World Hemophilia Day is April 17, and this year it will focus on the challenges of women and girls living with hemophilia and other bleeding disorders. The World Federation for Hemophilia (WFH) said the theme of its campaign is “Access for all: women and girls bleed too.”“Today, women and girls are still underdiagnosed and underserved,” the foundation says on its World Hemophilia Day website.
-
3 weeks ago | 
fabrydiseasenews.com | Marisa Wexler |Susanna VanVickle |Mary Chapman |Jerry Walter
For Fabry Awareness Month this April, the Fabry International Network, or FIN — an alliance of 61 patient organizations in 57 countries — is shining a spotlight on doctors who have focused on caring for people with the rare inherited disease, helping patients better understand the disorder, get accurate diagnoses, and access treatments. FIN has created a webpage highlighting the work of more than a dozen doctors worldwide who care for people with Fabry disease.
-
4 weeks ago | 
fabrydiseasenews.com | Lindsey Shapiro |Marisa Wexler |Steve Bryson |Mary Chapman
Distinct groups of inflammatory proteins may contribute to the progression of heart problems in men and women with Fabry disease, a study suggests. While men exhibited a profile associated with a strong pro-inflammatory response, women had a profile that could be linked to accumulating scar tissue. The identified markers may be useful for detecting cardiac problems in Fabry patients early and the findings suggest anti-inflammatory treatments may help slow cardiac progression.
-
1 month ago | 
hemophilianewstoday.com | Jacob Harney |Alliah Czarielle |Jennifer Lynne |Mary Chapman
March is Bleeding Disorders Awareness Month (BDAM), and patients, caregivers, advocates, and supporters are working together to call attention to and provide resources for coping with the bleeding disorders estimated to affect more than 3 million people in the U.S.Bleeding disorders such as hemophilia, von Willebrand disease, and rare clotting factor deficiencies are marked by the body’s inability to form blood clots.
-
Dec 10, 2024 | 
hemophilianewstoday.com | Patricia Inacio |Mary Chapman |Lindsey Shapiro |Margarida Maia
The first-in-human Phase 1/2 clinical trial testing BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B, is now enrolling patients at two sites in the U.S.BE-101, a first-in-class therapy, aims to address the persistent unmet needs of hemophilia B patients — particularly the burden of ongoing treatment and disease management for the “many people living with hemophilia B [who] continue to experience regular bleeding events,” Be Biopharma stated in a company press release.
-
Nov 19, 2024 | 
hemophilianewstoday.com | Andrea Lobo |Mary Chapman |Steve Bryson |Margarida Maia
Metagenomi, which is developing MGX-001, a gene-editing therapy for hemophilia A, is preparing to initiate investigational new drug (IND)-enabling activities that seek to support the therapy’s transition from preclinical to clinical testing. The move is backed by preclinical studies in nonhuman primates that show the treatment led to a sustained increase in the activity of factor VIII (FVIII), the missing clotting protein in hemophilia A, over a year.
-
Nov 11, 2024 | 
myastheniagravisnews.com | Shawna Barnes |Katherine Poinsatte |Sarah Bendiff |Mary Chapman
Do you prefer a window seat or an aisle seat when you travel? Before I got sick during my deployment to Iraq (2009-2010), I used to love to travel. I never had to give much thought to flying. I always went with the cheapest ticket or the best deal. When I was medically retired from the Army in 2011, I all but gave up the idea of traveling solo; my health was just too poor.
-
Oct 23, 2024 | 
myastheniagravisnews.com | Andrea Lobo |Shayna Korol |Lindsey Shapiro |Mary Chapman
The new UCB Myasthenia Gravis Scholarship, launched by the Belgium-based biopharmaceutical company, is aiming to help myasthenia gravis (MG) patients and their family members in the U.S. to continue their education or learn new skills by covering educational expenses — with program awards totaling up to $150,000.
-
Oct 15, 2024 | 
hemophilianewstoday.com | Andrea Lobo |Mary Chapman |Patricia Inacio |Lindsey Shapiro
The Spanish Interministerial Commission on the Pricing of Medicines, known as CIPM, has issued a positive recommendation for national reimbursement of Hemgenix (etranacogene dezaparvovec) for eligible people with hemophilia B, according to CSL Behring, the company that markets the gene therapy. The recommendation means that patients in Spain should soon have access to the gene therapy through the national healthcare system.
-
Oct 9, 2024 | 
ancavasculitisnews.com | Esteban Cerezo |Andrea Lobo |Allison Ross |Mary Chapman
The Vasculitis Foundation has announced the launch of a new support group for young adults with vasculitis, a group of autoimmune conditions that includes ANCA-associated vasculitis (AAV). The group will promote connections among young adults, from 18 to 30 years of age, living with vasculitis, which is marked by blood vessel inflammation.
