Thank you ALL for supporting last year’s sign on letter to add language to Newborn Screening Saves Lives Reauthorization which would fund states in order for every state to become RUSP compliant. As we know Newborn Screening Saves Lives Reauthorization is still undergoing challenges with pushback from Senator Rand Paul and the storage of dried …
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 …
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The ULF received donations in memorial or honorarium for the following during the month of May 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org or by calling 815.748.3211. IN HONOR OF Jacob: Nancy Reiners Jackson Jordan AJ Buckingham: Mr. & Mrs. Tony and Molly Hines …
Dylan was born on April 15, 2009, Daddy’s Little tax Deduction! For the first 5 months he progressed like a normal child, until one day, which seemed like a case of collie, he was taken into Children’s Hospital of Pittsburgh, by his new, first time mom. The visit proved to be anything than routine. After …
From PatientWorthy.com After coming back from the Global Genes conference, Kristie was convinced that establishing a foundation was the right next step to advocate for a cure for Refsum Disease. With the help of other patients and family, Global DARE Foundation was formed with 5 board members. DARE stands for Defeat Adult Refsum Everywhere. The first board …
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 »
The ULF received donations in memorial or honorarium for the following during the month of April 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org or by calling 815.748.3211. IN HONOR OF Ryan D. Miller: Ellen Dodaro Daxton Wallace: Sandy Wallace IN MEMORY OF Joseph Abelman: …
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 …
