Finding Strength in Community: Jen’s Story
I’m Jen Martin-Cutler, proud mom to Clark, my handsome ginger son diagnosed with Pelizaeus-Merzbacher Disease (PMD) in December 2015 at 11 months old. Clark’s leukodystrophy journey began with challenges like delayed milestones, difficulty swallowing, and nystagmus, leading us to specialists at Lurie Children’s Hospital. His care team included experts in genetics,…
Living with ALD: A Journey of Strength and Community
My name is Margaret Halter. My husband, John, was diagnosed with Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN) in 1989 at the age of 31. We’ve been married for 41 years, live on a beautiful farm in Ohio, and have two children, a daughter and a son, both with families of their own. Our daughter…
Minoxry Therapeutics: Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cALD
Press Release Summary (December 11, 2024): Minoryx Therapeutics, a late-stage biotech company, and Neuraxpharm Group, a European specialty pharmaceutical firm, announced positive outcomes from the NEXUS trial, a pivotal 96-week study evaluating leriglitazone in pediatric patients with cerebral Adrenoleukodystrophy (cALD). The trial demonstrated a significant 35% arrest of disease progression…
A Special Year-End Message from Dr. Mousumi Bose
My name is Mousumi Bose, and I am the mother of Ilan Betzer. Ilan was born in August 2010 and was soon diagnosed with Zellweger spectrum disorder, a rare peroxisomal disorder that is categorized as a leukodystrophy. As his disease progressed, our journey with Ilan was marked by complex challenges. Ilan passed…
Help Us Bring Hope to Families Like Isabella’s This Holiday Season
I’m the aunt of my beautiful niece, Isabella, who was diagnosed with Vanishing White Matter (VWM) Disease at age four in 2009. Isabella’s joyful spirit and incredible courage inspire me to be the best I can be. She faces the challenges of this devastating condition with a smile that never…
Beverly’s Journey of Hope & Giving Back
“I was so fortunate to have been able to attend the ULF conference without worrying about finances, and other families should not have to be concerned about costs to be there.” Beverly Jones Devoted mother & longtime ULF supporter Beverly Jones, a devoted mother, first connected with the United Leukodystrophy…
In Memorial and Honor: August – September 2024
ULF received donations in memorial or honorarium for the following during the months of August through September 2024. 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 Clark Cutler Jennifer Martin-Cutler Saddle up…
Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease
Aspa Therapeutics: A Natural History Study of Patients With Canavan Disease (CANinform Study) [https://clinicaltrials.gov/study/NCT04126005] CANinform is a natural history study of patients with Canavan disease that will help researchers better understand the disease and provide a foundation for Aspa’s gene therapy program. The study is open to individuals with a confirmed diagnosis of Canavan disease.
BridgeBio press release about the RMAT designation for the Aspa clinical trial in Canavan Disease
09.10.2024 at 4:45 PM EDT – Receipt of RMAT Designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which showed functional improvements in all dosed patients indicating that BBP-812 has potential to address the unmet needs of individuals with Canavan disease – BridgeBio will leverage…
CTX Patient Assistance Program is open and accepting assistance applications
NORD’s Cerebrotendinous Xanthomatosis (CTX) Patient Assistance Program is open and accepting applications for assistance The quickest way to apply is on NORD’s website at: bit.ly/patient-assist. Scroll down to Cerebrotendinous Xanthomatosis (CTX) and click Apply Online.
In Memorial and Honor: May – July 2024
ULF received donations in memorial or honorarium for the following during the months of May through July 2024. 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 Dan Mulligan Troy Glendenning Tara Nunez…
An Update from Passage Bio on Out-Licensing of Pediatric Programs to GEMMA Biotherapeutics
On August 1, 2024, Passage Bio shared they entered into a new agreement with GEMMA Biotherapeutics for the out-licensing of three pediatric gene therapy programs. Through this transaction, Passage Bio has granted GEMMA Biotherapeutics the rights for the development and commercialization of PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease,…
In Memorial and Honor – April 2024
ULF received donations in memorial or honorarium for the following during the month of April 2024. 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 Brad & Lance Yotti James Bowersett Sue Bowersett…
In Memorial and Honor – March 2024
ULF received donations in memorial or honorarium for the following during the month of March 2024. 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 BRAMBLEBOX TURTLE Melissa Kapoor Daxton Wallace Sandra Wallace…
Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy
The ULF is thrilled to share this press release from Orchard Therapeutics regarding their new FDA approved therapy for MLD! “Lenmeldy is truly a paradigm-shifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment, particularly when administered prior to…
FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy
The ULF is shared to be able to share this press release from the Food & Drug Association: “Today, the U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic…
In Memorial and Honor – February 2024
ULF received donations in memorial or honorarium for the following during the month of February 2024. 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 Adele Moss Claudette Alice Collier Benjamin Rosenberg Ari…
Two ALSP Studies Now Recruiting
The ULF is happy to share this information on behalf of Vigil Neuro. Contact information is listed below for more information. Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP) A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia If…
In Memorial and Honor – January 2024
ULF received donations in memorial or honorarium for the following during the month of January 2024. 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 Daxton Wallace Sandra Wallace Kenan Spencer Witczak Jennifer…
Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote
The ULF is happy to share this press release on behalf of Forge Biologics regarding their clinical trial for patients with Krabbe. Here is a preview but click the link below to read the full press release. UPDATE: As of Tuesday, January 30: The ACHDNC has recommended the addition of…
In Memorial and Honor – December 2023
ULF received donations in memorial or honorarium for the following during the month of December 2023. 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 Adam Sledzik Alicja Sledzik AJ Woodard Anonymous Alec…
Daniil’s Story
Daniil Zamorin wrote this story, but is a native Russian speaker. The ULF has helped Daniil edit his story to be more easily understood by an English-speaking audience. He welcomes people to reach out to him, and the ULF would be happy to facilitate the connection. Please email [email protected] to…