Shortcast: Liver Directed Rx don’t change biochemistry nor Leukodystrophy in Biallelic HMBS Variants

from JIMD Podcasts · host Journal of Inherited Metabolic Disease

Dr Jeremy Clark unpacks why leukodystrophy caused by biallelic HMBS variants does not respond to liver transplantation or hepatically targeted therapies, pointing instead to CNS-driven porphyrin toxicity and a need for entirely new management approaches. Liver Transplantation and Other Hepatically Directed Therapies Do Not Change the Biochemical Phenotype nor Halt Progression of Leukodystrophy due to Biallelic HMBS Variants: A Case Report Jeremy Clark, et al https://doi.org/10.1002/jmd2.70056

Episode metadata supplied by the publisher feed · Published Dec 30, 2025

Embed this episode

Attribution link and audio player

NOW PLAYING

Shortcast: Liver Directed Rx don’t change biochemistry nor Leukodystrophy in Biallelic HMBS Variants

0:00 4:23

1×

No transcript for this episode yet

We transcribe on demand. Request one and we'll notify you when it's ready — usually under 10 minutes.

Share this episode

Similar Episodes

No similar episodes found.

Similar Podcasts

No similar podcasts found.

Frequently Asked Questions

How long is this episode of JIMD Podcasts?

This episode is 4 minutes long.

When was this JIMD Podcasts episode published?

This episode was published on December 30, 2025.

Can I download this JIMD Podcasts episode?

Yes. Use the download control on the episode player to save the publisher-provided media file.

URL copied to clipboard!