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June 9, 2026Neurology

GHF-201, A Novel Oral Compound for Adult Polyglucosan Body Disease, Demonstrates Promising Safety and Efficacy in a Compassionate Use Program (S2.010)

Or Kakhlon Anat Mordechai Nissim Garti Yossi Gilgun‐Sherki Miguel Weil Alexander Lossos

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Key Points

To evaluate the safety, tolerability, and efficacy of GHF-201 in patients with Adult Polyglucosan Body Disease.
Compassionate use program involving 3 patients diagnosed with Adult Polyglucosan Body Disease.
Assessments focused on safety and tolerability alongside efficacy outcomes.
GHF-201 exhibited promising safety profiles with no significant adverse effects reported.
Efficacy measures showed improvement in symptoms related to Adult Polyglucosan Body Disease.

Implication

Demonstrates safety and efficacy of GHF-201 in patients with Adult Polyglucosan Body Disease, suggesting a potential treatment option.

Abstract

Assess the safety, tolerability and efficacy of GHF-201 in 3 patients with Adult Polyglucosan Body Disease (APBD) in a Compassionate Use program.

Discussion

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Cite This Study

Kakhlon et al. (2026) studied this question.

synapsesocial.com/papers/6a2cf3b4faef96ed7f0561ad https://doi.org/10.1212/wnl.0000000000215856

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