The novel coronavirus pandemic can have a serious impact on people with rare diseases and their families. Yet, not much is known about it. We want to learn from you and hope that you are willing to share your experience. The Rare Diseases Clinical Research Network’s (RDCRN) Data Management and Coordinating Center, which is supported …
NORD invites you to register for an externally-led Patient-Focused Drug Development (EL- PFDD) virtual meeting on Krabbe disease, taking place Thursday, October 29, 12:30 – 4:30 pm ET. Patients and caregivers impacted by Krabbe disease who are interested in potentially speaking at the meeting as panelists should be sure to fill out this form by August 7. If you …
Patient Focused Drug Development Meeting on Krabbe Disease Read More »
from BlueCross BlueShield of Illinois Look around and you’ll see caregivers. In the U.S., 1 in 3 adults give care to others. Caregivers may be taking care of elderly parents, a sick spouse or a disabled child. Being a caregiver can be very stressful and demanding. It can even lead to caregiver burnout, a feeling …
Taking Care of Yourself When Taking Care of Others Read More »
Minoryx Therapeutics is recruiting for a clinical trial focusing on cALD in Europe. The NEXUS clinical trial is a phase 2, open-label clinical study designed to assess the efficacy and safety of leriglitazone in male pediatric patients with early stage cerebral X-linked adrenoleukodystrophy (cALD). The NEXUS study recruits cALD patients with early cerebral MRI lesions. …
Join the Global Day of Giving and Unity! #GivingTuesdayNow is set to take place on May 5, 2020 as an emergency response to the unprecedented need caused by COVID-19. The day is designed to drive an influx of generosity, citizen engagement, business and philanthropy activation, and support for communities and nonprofits around the world. At …
by The Montreal Children’s Hospital Foundation Dr. Geneviève Bernard is a world-renowned expert on genetically determined white matter disorders, called leukodystrophies. Patients are referred to her clinic from all over the world. She brings much comfort to families everywhere who are suffering from these diseases for which the vast majority currently have no known cure. …
Life During the Covid-19 Crisis: A Chat with Dr. Bernard Read More »
March 2020 Dear ULF Community, On behalf of the Board of Directors, Medical and Scientific Advisory Board, and the staff of the ULF, please know that we are following the developments of COVID-19 and feel the concerns of our community. At this time, all we can offer is advice on staying healthy and safe during …
Dear Friends and Supporters of the United Leukodystrophy Foundation, As the President of the ULF I am excited to talk to you about the many changes that are taking place as we build the future of the Foundation. In the past year, we’ve added seven new members to our board of directors. These new additions …
Tickets are $50 each, and only 500 tickets will be sold. You are welcome to purchase as many tickets as you would like. Payment must be received by 12 PM Noon CST on Friday, March 27th, 2020. The drawing will be streamed on Facebook Live at 2 PM CST on the 27th. Follow us on …
Tickets for the 3rd Annual Magical Memories Disney Raffle are now available! Read More »
Worldwide, MLD occurs in 1 in 40,000 people, primarily children. Early symptoms of MLD include trouble walking, impaired speech, and muscle weakness. The Embolden Study is conducting research for infants and children born with metachromatic leukodystrophy. Study overview: The Embolden Study is evaluating the safety and effects of a study drug on gross motor function (the abilities …
