Year: 2024

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 the onset of symptoms,” said …

Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy Read More »

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 early juvenile metachromatic leukodystrophy (MLD).” …

FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy Read More »

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 you are interested in learning …

Two ALSP Studies Now Recruiting Read More »

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 Krabbe disease to the RUSP! …

Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote Read More »