Correction: Expert Group Consensus on early diagnosis and management of infantile-onset pompe disease in the Gulf Region
- Location
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Deutsche Nationalbibliothek Frankfurt am Main
- Extent
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1 Online-Ressource.
- Language
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Englisch
- Bibliographic citation
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Correction: Expert Group Consensus on early diagnosis and management of infantile-onset pompe disease in the Gulf Region ; volume:18 ; number:1 ; day:23 ; month:11 ; year:2023 ; pages:1 ; date:12.2023
Orphanet journal of rare diseases ; 18, Heft 1 (23.11.2023), 1, 12.2023
- Creator
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Al-Hassnan, Zuhair
Al Hashmi, Nadia
Makhseed, Nawal
Omran, Tawfeg Ben
Al Jasmi, Fatma
Al Teneiji, Amal
- Contributor
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SpringerLink (Online service)
- DOI
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10.1186/s13023-023-02967-0
- URN
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urn:nbn:de:101:1-2024021309031563883302
- Rights
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Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Last update
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15.08.2025, 7:32 AM CEST
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Associated
- Al-Hassnan, Zuhair
- Al Hashmi, Nadia
- Makhseed, Nawal
- Omran, Tawfeg Ben
- Al Jasmi, Fatma
- Al Teneiji, Amal
- SpringerLink (Online service)
Other Objects (12)
Expert Group Consensus on early diagnosis and management of infantile-onset pompe disease in the Gulf Region
Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy
Infantile Pompe disease with intrauterine onset: a case report and literature review
Clinical course, mutations and its functional characteristics of infantile-onset Pompe disease in Thailand
Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry
Infantile-onset pompe disease: a case report emphasizing the role of genetic counseling and prenatal testing
An expert rule-based approach for identifying infantile-onset Pompe disease patients using retrospective electronic health records
Analyzing immune cell infiltrates in skeletal muscle of infantile-onset Pompe disease using bioinformatics and machine learning
CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease
Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
Slow, progressive myopathy in neonatally treated patients with infantile-onset Pompe disease: a muscle magnetic resonance imaging study
Cognitive outcome of classic infantile Pompe patients receiving enzyme therapy
Expert Group Consensus on early diagnosis and management of infantile-onset pompe disease in the Gulf Region
Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy
Infantile Pompe disease with intrauterine onset: a case report and literature review
Clinical course, mutations and its functional characteristics of infantile-onset Pompe disease in Thailand
Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry
Infantile-onset pompe disease: a case report emphasizing the role of genetic counseling and prenatal testing
An expert rule-based approach for identifying infantile-onset Pompe disease patients using retrospective electronic health records
Analyzing immune cell infiltrates in skeletal muscle of infantile-onset Pompe disease using bioinformatics and machine learning
CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease
Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
Slow, progressive myopathy in neonatally treated patients with infantile-onset Pompe disease: a muscle magnetic resonance imaging study
Cognitive outcome of classic infantile Pompe patients receiving enzyme therapy
Expert Group Consensus on early diagnosis and management of infantile-onset pompe disease in the Gulf Region
Hearing characteristics of infantile-onset Pompe disease after early enzyme-replacement therapy
Infantile Pompe disease with intrauterine onset: a case report and literature review
Clinical course, mutations and its functional characteristics of infantile-onset Pompe disease in Thailand
Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry
Infantile-onset pompe disease: a case report emphasizing the role of genetic counseling and prenatal testing
An expert rule-based approach for identifying infantile-onset Pompe disease patients using retrospective electronic health records
Analyzing immune cell infiltrates in skeletal muscle of infantile-onset Pompe disease using bioinformatics and machine learning
CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease
Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy
Slow, progressive myopathy in neonatally treated patients with infantile-onset Pompe disease: a muscle magnetic resonance imaging study