Leukodystrophy

Neurology·MeSH: Leukodystrophy, Metachromatic

Prevalence

0.5 / 100K

8 drugs in development·5 companies·2 clinical trials

Drug Pipeline by Phase

Approved(2)

Drug	Company
libmeldy	Orchard Therapeutics plc (ORTX)
SKYSONA	bluebird bio, Inc. (BLUE)

Phase 2(2)

Drug	Company
otl-200	Orchard Therapeutics plc (ORTX)
shp611	SHIRE PLC (SHPGF)

Phase 1(1)

Drug	Company
VK0214	Viking Therapeutics, Inc. (VKTX)

Phase 1/Phase 2(3)

Drug	Company
hgt-1110	SHIRE PLC (SHPGF)
otl-200 gene therapy	Orchard Therapeutics plc (ORTX)
pbkr03	IMMAGE BIOTHERAPEUTICS CORP. (IMMG)

BioSniper tracks 8 drugs in development for Leukodystrophy across 5 companies.

There are 2 clinical trials targeting Leukodystrophy tracked by BioSniper.

The estimated prevalence of Leukodystrophy is approximately 0.5 per 100,000 population.

Full trial data, competitive analysis, and AI-powered pipeline tracking.