In Memorial or Honorarium – April 2021
The ULF received donations in memorial or honorarium for the following during the month of April 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Aidan ChapleauRobert KiddBarry MacDonaldMarg MacDonald All those fighting leukodystrophies…
Axel Riordan, MLD
By Alexandra Worth Axel Riordan was born October 15, 2017. He was a healthy growing boy, hitting all his milestones on time. Around 18 months old Axel started developing problems while walking; locking knees, stumbling, eventually falling a lot. It all progressed slowly at first so at his two-year checkup…
In Memorial or Honorarium – March 2021
The ULF received donations in memorial or honorarium for the following during the month of March 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Alec JumpJames Jump Leesa Jump Alex Bouley Charna Levine Brad…
Shari S., ALD
Being part of a family that has endured the devastation of ALD since 1959 when my mother’s brother lost his battle and then losing both my older brother in 1972 and my younger brother in 1979. Knowing since 1987 when I was 18 years old that I am a carrier set…
In Memorial or Honorarium – February 2021
The ULF received donations in memorial or honorarium for the following during the month of February 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Henry Crawley David Crawley – I love you Henry. …
Amera Reed, ACER3
by Shannon Reed Amera is our sweet 3 year old daughter who battles an extremely rare genetic disease called ACER3 related early childhood onset progressive Leukodystrophy. There are only about 20 known cases of this type worldwide! Our daughter was diagnosed with this progressive disease at age 2. She was…
In Memorial or Honorarium – January 2021
The ULF received donations in memorial or honorarium for the following during the month of January 2021. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Aryabella Haeger Shannon Wright – In behalf of Star…
We’re All in This Together #RareDiseaseDay
In honor of Rare Disease Day, we have come together to raise funds for type-specific programming. This shirt sale is made possible by a collaborative effort among these amazing organizations that will help offset future costs of type-specific programming. We are all in this together. Orders will be shipped directly…
Orchard Therapeutics Announces OTL-200 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation by FDA for the Treatment of Metachromatic Leukodystrophy (MLD)
Interactions with FDA anticipated by mid-2021 to determine the BLA filing strategy for OTL-200 BOSTON and LONDON, Jan. 14, 2021 (GLOBE NEWSWIRE) — Orchard Therapeutics, a global gene therapy leader, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OTL-200,…
In Memorial or Honorarium – December 2020
The ULF received donations in memorial or honorarium for the following during the month of December 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Aayaansh ModiAnonymous Ainsley CunninghamAnonymous Cheryl Malden Alex Hiles Cynthia Powell …
FDA Clearance of Investigational New Drug for Patients with Krabbe Disease
January 4, 2021 by Dan Salvo Forge Biologics has received IND, IBC, and IRB clearance for FBX-101, a gene therapy for patients with Krabbe disease, a rare and fatal genetic disorder FBX-101 is a first-in-human potential treatment utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene to…
Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
Press Release: Orchard Therapeutics BOSTON/LONDON/MILAN, December 21, 2020 – Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene),…
In Memorial or Honorarium – November 2020
The ULF received donations in memorial or honorarium for the following during the month of November 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Alex Bouley Bernard Levine, M.D. Aryabella HaegerShannon Wright Daxton…
In Memorial or Honorarium – October 2020
The ULF received donations in memorial or honorarium for the following during the month of October 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF C. Michael Jaynes: Angela Pontius, C. Michael Jaynes –…
In Memorial or Honorarium – September 2020
The ULF received donations in memorial or honorarium for the following during the month of September 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Ella McKee: Heather McKee – For our brave daughter…
In Memorial or Honorarium – August 2020
The ULF received donations in memorial or honorarium for the following during the month of August 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Amie Matlon: Marianne Matlon Aveline Brendel: Pauline Naudin, William…
In Memorial or Honorarium – July 2020
The ULF received donations in memorial or honorarium for the following during the month of July 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF David Stocki: Melissa Scier – For my son who…
In Memorial or Honorarium – June 2020
The ULF received donations in memorial or honorarium for the following during the month of June 2020. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at [email protected] or by calling 815.748.3211. IN HONOR OF Angelica Gallegos: Rozzana Vasquez Cobos Anna Walentowski: Anonymous Briana…
Newborn Screening Saves Lives Reauthorization
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…
Research Study: Impact of COVID-19 on People Living with Rare Diseases and Their Families
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…
Patient Focused Drug Development Meeting on Krabbe Disease
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…
In Memorial or Honorarium – May 2020
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 [email protected] or by calling 815.748.3211. IN HONOR OF Jacob: Nancy Reiners Jackson Jordan AJ Buckingham: Mr. &…
Dylan Joseph Changle, Krabbe Disease
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…
Kristie DeMarco, Refsum Disease
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…
Taking Care of Yourself When Taking Care of Others
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…