Please provide your email address to receive an email when new articles are posted on . Biotech firm SynaptixBio has entered into a licensing agreement with the Children’s Hospital of Philadelphia, ...
A new medical treatment that costs more than $4 million for metachromatic leukodystrophy (MLD) was approved recently by the FDA. MLD was recognized as a disease in the early 1900s, although specifics ...
The global market was valued at USD 3.23 Billion in 2022 and is expected to reach USD 3.23 Billion by 2032, registering a CAGR of 4.8% from 2023 to 2032. PUNE ...
Clin Lipidology. 2009;4(2):205-213. The conditioning or preparative program for HSCT typically involves a combination of irradiation and chemotherapy to completely destroy the host bone marrow (i.e., ...
In metachromatic leukodystrophy (MLD), the deficiency of the lysosomal enzyme arylsulfatase A (ARSA) leads to demyelination in the central and peripheral nervous system and ultimately to death.
LYON, France--(BUSINESS WIRE)--POXEL SA (Euronext : POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA approved ...
"Metachromatic Leukodystrophy Pipeline 2025"DelveInsight's,“Metachromatic Leukodystrophy - Pipeline Insight, 2025,” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in ...
Leukodystrophies are a heterogeneous group of inherited disorders with highly variable clinical manifestations and pathogenetic background Leukodystrophies are characterized by primary glial cell and ...
The evidence presented above document a role for VLCFAs in peroxisomal dysfunction that lead to elevated ROS, oxidative stress and the subsequent inflammatory demyelination observed in ALD patients.