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 office@ulf.org or by calling 815-748-3211 In Honor of Clark Cutler Jennifer Martin-Cutler Saddle up – Kicks for clark event. …
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) Study Overview Brief Summary The primary purpose of this study is to evaluate the safety and tolerability of ION356. Detailed Description This is a Phase 1b, open-label multiple-ascending dose (MAD) study of ION356 …
Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease (CANaspire Clinical Trial) [https://treatcanavan.com/canaspire-trial/] Aspa’s investigational gene therapy trial uses an AAV9 vector (adeno-associated virus serotype 9), which is designed to deliver functional copies of the ASPA gene throughout the body, including into the brain, to address the underlying cause of Canavan disease. The AAV9 …
Aspa Therapeutics: A Study of AAV9 Gene Therapy in Children With Canavan Disease Read More »
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.
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 the benefits of RMAT designation, …
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.
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 office@ulf.org or by calling 815-748-3211 In Honor of Dan Mulligan Troy Glendenning Tara Nunez Quantum Crossing, LLC Master Built …
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, and PBML04 for metachromatic leukodystrophy. …
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 office@ulf.org or by calling 815-748-3211 In Honor of Brad & Lance Yotti James Bowersett Sue Bowersett Daxton Wallace Sandra Wallace Mia …
