๐๏ธ New JIMD Shortcast
Leukodystrophy caused by biallelic HMBS variants behaves very differently from acute intermittent porphyria and does not respond to liver-directed therapies.
๐ง Listen now: open.spotify.com/episode/5qWM...
#RareDisease #Leukodystrophy #Porphyria #MetabolicNeurology #IMD
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