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Update on the MLD NBS RUSP Nomination – and Newborn Screening in General

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We’re still moving the MLD RUSP nomination forward. We gave/discussed this update at two NBS meetings last week, on an EveryLife Foundation Community Congress ad-hoc call, and in numerous conversations with other patient advocacy groups and bio-pharma companies.  We don’t want to leave the MLD community and families out of the update loop, hence this […]

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Dean & Teryn Suhr are presented with the WORLDsymposium™ 2025 PAL award

Dean & Teryn Suhr Recognized With the 2025 Patient Advocacy Leadership (PAL) Award​ at WORLDsymposium™

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On Wednesday, Feb 5th, 2025 Dean & Teryn Suhr, co-Founders of MLD Foundation, were awarded the 2025 PAL (Patient Advocacy Leadership) Award by from WORLDsymposium™, a consortium of over 1800 lysosomal disease researchers,  academia, institutional,  biotech, and advocacy leaders. A sincere congratulations to Dean & Teryn Suhr, the recipient of the esteemed WORLDSymposium 2025 Patient

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FDA BLA Filing Completed for MLD Gene Therapy

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FDA Filing Completed for MLD Gene Therapy Orchard Therapeutics requesting Priority Review of BLA Orchard Therapeutics has announced the completion of their BLA filing and a request for Priority Review with the US FDA for OTL-200, the gene therapy approved as Libmeldy in Europe.  Their “rolling-BLA” filings started last quarter. The BLA is a “Biologics License Application“, the process used to get an

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Takeda announces preliminary ERT trial results

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Takeda announces preliminary ERT trial results Early results not as positive as hoped for Takeda Pharmaceuticals announced that a preliminary look at the results of their recently completed phase-IIb trial of their TAK-611 ERT (enzyme replacement therapy) for MLD “topline results … did not meet primary and secondary endpoints.” The company is analyzing the results and

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Orchard Starts Filings with FDA for MLD Gene Therapy

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Orchard Starts FDA MLD Gene Therapy Filings Orchard Therapeutics has announced they have filed the first module of their rolling BLA  with the US FDA  for OTL-200, the gene therapy approved as Libmeldy in Europe.  The BLA is a “Biologics License Application” that will be reviewed by the FDA. A “rolling BLA” allows Orchard to file the application in multiple parts, which

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Affinia Therapeutics Announces MLD Program

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Today MLD Foundation welcomes Affinia Therapeutics to the MLD Family™! Affinia is a US-based biopharma startup working on a new approach to MLD gene therapy. We are excited to share that MLD is their first therapy program! Affinia Therapeutics’ product candidate Anc80L65-ARSA is designed to deliver a functional ARSA transgene via a one-time dose to

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