Hassnan ZA, Hashmi NA, Makhseed N, Omran TB, Al Jasmi F et al.

Expert Group Consensus on early diagnosis and management of infantile-onset pompe disease in the Gulf Region.

Orphanet J Rare Dis. 2022 ;17:388. 

Hahn A, Schänzer A.

Long-term outcome and unmet needs in infantile-onset Pompe disease.

Ann Transl Med. 2019 ;7:283

Ditters IAM, Huidekoper HH, Kruijshaar ME, Rizopoulos D, Hahn A et al ;

European Pompe Consortium project group on classic infantile Pompe disease. Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.

Lancet Child Adolesc Health. 2022 ;6:28-37