Current diagnosis and management of rare pediatric diseases in China

Yang D, Ren X, Lu Y, Han J

Intractable Rare Dis Res. 2021; 10(4):223-237.

DOI: 10.5582/irdr.2021.01134

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Off-label medication use in rare pediatric diseases in the United States

Fung A, Yue X, Wigle PR, Guo JJ

Intractable Rare Dis Res. 2021; 10(4):238-245.

DOI: 10.5582/irdr.2021.01104

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Effect of nutritional intervention on nutritional status among children with disorders of amino acid and nitrogen metabolism (AANMDs): A scoping review

Lim JY, Amit N, Ali NM, Leong HY, Mohamad M, Rajikan R

Intractable Rare Dis Res. 2021; 10(4):246-256.

DOI: 10.5582/irdr.2021.01124

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A review of Alström syndrome: a rare monogenic ciliopathy

Roy Choudhury A, Munonye I, Sanu KP, Islam N, Gadaga C

Intractable Rare Dis Res. 2021; 10(4):257-262.

DOI: 10.5582/irdr.2021.01113

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Effects of a personalized home-based training program among patients suffering from Marfan syndrome: a pilot randomized and controlled study

Jouini S, Milleron O, Eliahou L, Jondeau G, Vitiello D

Intractable Rare Dis Res. 2021; 10(4):263-268.

DOI: 10.5582/irdr.2021.01080

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Alteration of skeletal and cardiac muscles function in DBA/2J mdx mice background: a focus on high intensity interval training

Baati N, Mougenot N, Lemaitre M, Kirsch M, Agbulut O, Ferry A, Vitiello D

Intractable Rare Dis Res. 2021; 10(4):269-275.

DOI: 10.5582/irdr.2021.01097

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Serum neurofilament light chain is not a useful biomarker of central nervous system involvement in women with Fabry disease

Hołub T, Kędzierska K, Muras-Szwedziak K, Nowicki M

Intractable Rare Dis Res. 2021; 10(4):276-282.

DOI: 10.5582/irdr.2021.01115

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Current status of specific pediatric chronic diseases in Japan: National measures, disease types, treatment availability, copayment assistance, and research

Sawakami T

Intractable Rare Dis Res. 2021; 10(4):283-287.

DOI: 10.5582/irdr.2021.01145

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Updated genetic studies of Marfan syndrome in China

Sun YX, Zhou D, Wang SH, Ding J, Ma F

Intractable Rare Dis Res. 2021; 10(4):288-291.

DOI: 10.5582/irdr.2021.01139

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GPR30: A new potential therapeutic target in human testicular germ cell tumors

Chieffi P

Intractable Rare Dis Res. 2021; 10(4):292-293.

DOI: 10.5582/irdr.2021.01103

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