News

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…

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…

ALSP Community Survey: Genetic Testing

Vigil Neuroscience, a biotechnology company researching potential treatments for ALSP, is engaging with the ALSP patient community, caregivers, advocacy groups, and healthcare providers to better understand the needs of community members.  Open Health is an independent company engaged by Vigil Neuroscience to conduct a survey to understand behaviors and attitudes towards genetic testing for ALSP…