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Living with Pompe

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FDA clears Pombiliti plus Opfolda for certain adults with LOPD

Pompe Disease News
In Pompe, infusion-associated reactions to ERT mostly mild...

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Pompe patients like infusing enzyme replacement therapy at home

Pompe Disease News
Pompe gene therapy able to correct protein defects in mice: Study

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Study sheds light on how SMN protein deficiency can cause SMA

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Late-onset Pompe severity varied widely in gene therapy screening...

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Pompe disease symptoms

Resources about Pompe Disease
ABBV-951 now available at low/no cost in England to some patients

Science & Research News: Parkinson's Disease
Gene Therapy and Pompe Disease

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3 Pompe siblings have same mutations, different symptoms: Report

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How Is Pompe Disease Diagnosed

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Late-onset Pompe disease

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