uncategorized

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 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 »

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 early juvenile metachromatic leukodystrophy (MLD).” …

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

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 you are interested in learning …

Two ALSP Studies Now Recruiting Read More »

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 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 »

Minoryx Announces Patient Enrollment of their CALYX Trial

The ULF is happy to share this press release on behalf of Minoryx regarding their CALYX Trial for patients affected by cALD. Here is a preview but click the link below to read the full press release. “Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system …

Minoryx Announces Patient Enrollment of their CALYX Trial Read More »

The United Leukodystrophy Foundation Welcomes New Executive Director

FOR IMMEDIATE RELEASE The United Leukodystrophy Foundation Welcomes New Executive Director DeKalb, IL – The United Leukodystrophy Foundation (ULF) is pleased to announce the appointment of Diane Fennimore as its new Executive Director. Fennimore is a seasoned business development executive with more than twenty years of leadership experience in mission-driven organizations. She has a deep …

The United Leukodystrophy Foundation Welcomes New Executive Director Read More »

Poxel Announces New Strategic Direction with Increasing Focus on Rare Metabolic Diseases Following Recent Achievements

Download as PDF July 12, 2021 Following the recent approval of TWYMEEG® (Imeglimin) in Japan and associated potential future revenues, Poxel to accelerate and expand rare metabolic disease programs leveraging existing platforms and proven capabilities Poxel to advance its deuterated thiazolidinediones (dTZD) and direct adenosine monophosphate-activated protein kinase (AMPK) activator platforms in rare metabolic diseases …

Poxel Announces New Strategic Direction with Increasing Focus on Rare Metabolic Diseases Following Recent Achievements Read More »

FDA Clearance of Investigational New Drug for Patients with Krabbe Disease

January 4, 2021 by Dan Salvo Forge Biologics has received IND, IBC, and IRB clearance for FBX-101, a gene therapy for patients with Krabbe disease, a rare and fatal genetic disorder FBX-101 is a first-in-human potential treatment utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene to cells in the CNS and …

FDA Clearance of Investigational New Drug for Patients with Krabbe Disease Read More »