Evaluating psychosocial stressors in families of children with inborn errors of metabolism von Grant, Nathan, Burrow, T. Andrew

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Correlating liver stiffness with disease severity scoring system (DS3) values in Gaucher disease type 1 (GD1) patients von Serai, Suraj D., Naidu, Anjani P., Andrew Burrow, T., Prada, Carlos E., Xanthakos, Stavra, Towbin, Alexander J.

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Cardiac Disease in Methylmalonic Acidemia von Prada, Carlos E., MD, Al Jasmi, Fatma, MD, Kirk, Edwin P., MB BS, Hopp, Maxwell, MB BCh, Jones, Owen, MB BChir, Leslie, Nancy D., MD, Burrow, T. Andrew, MD

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Acute Progression of Neuromuscular Findings in Infantile Pompe Disease von Burrow, T. Andrew, MD, Bailey, Laurie A., MS, Kinnett, Douglas G., MD, Hopkin, Robert J., MD

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Profound hypotonia in an infant with δ-aminolevulinic acid dehydratase deficient porphyria von Roach, Alexis N, Barkley, Hannah, Rodriquez, Carissa, Burrow, T Andrew, Anderson, Karl E, Shukla, Ankita

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The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here? von Weinreb, Neal J., Goker-Alpan, Ozlem, Kishnani, Priya S., Longo, Nicola, Burrow, T. Andrew, Bernat, John A., Gupta, Punita, Henderson, Nadene, Pedro, Helio, Prada, Carlos E., Vats, Divya, Pathak, Ravi R., Wright, Ekaterina, Ficicioglu, Can

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Gaucher disease and SARS-CoV-2 infection: Emerging management challenges von Mistry, Pramod, Balwani, Manisha, Barbouth, Deborah, Burrow, T. Andrew, Ginns, Edward I., Goker-Alpan, Ozlem, Grabowski, Gregory A., Kartha, Reena V., Kishnani, Priya S., Lau, Heather, Lee, Chung U., Lopez, Grisel, Maegawa, Gustavo, Packman, Seymour, Prada, Carlos, Rosenbloom, Barry, Lal, Tamanna Roshan, Schiffmann, Rapheal, Weinreb, Neal, Sidransky, Ellen

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Diagnosis and management of glycogen storage disease type IV, including adult polyglucosan body disease: A clinical practice resource von Koch, Rebecca L., Soler-Alfonso, Claudia, Kiely, Bridget T., Asai, Akihiro, Smith, Ariana L., Bali, Deeksha S., Kang, Peter B., Landstrom, Andrew P., Akman, H. Orhan, Burrow, T. Andrew, Orthmann-Murphy, Jennifer L., Goldman, Deberah S., Pendyal, Surekha, El-Gharbawy, Areeg H., Austin, Stephanie L., Case, Laura E., Schiffmann, Raphael, Hirano, Michio, Kishnani, Priya S.

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Squalene Synthase Deficiency: Clinical, Biochemical, and Molecular Characterization of a Defect in Cholesterol Biosynthesis von Coman, David, Vissers, Lisenka E.L.M., Riley, Lisa G., Kwint, Michael P., Hauck, Roxanna, Koster, Janet, Geuer, Sinje, Hopkins, Sarah, Hallinan, Barbra, Sweetman, Larry, Engelke, Udo F.H., Burrow, T. Andrew, Cardinal, John, McGill, James, Inwood, Anita, Gurnsey, Christine, Waterham, Hans R., Christodoulou, John, Wevers, Ron A., Pitt, James

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CNTNAP1-Related Congenital Hypomyelinating Neuropathy von Lesmana, Harry, Vawter Lee, Marissa, Hosseini, Seyed Ali, Burrow, T. Andrew, Hallinan, Barbara, Bove, Kevin, Schapiro, Mark, Hopkin, Robert J.

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Clinical insights from Wolman disease: Evaluating infantile hepatosplenomegaly von Hannah, William B., Ryan, Katherine, Pendyal, Surekha, Burrow, T. Andrew, Harley, Susan E., Cordell, Miranda, McCall, Chad M., Mavis, Alisha M., Tan, Queenie K.‐G., Kishnani, Priya S.

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Gaucher Disease: Progressive Mesenteric and Mediastinal Lymphadenopathy Despite Enzyme Therapy von Burrow, T. Andrew, MD, Cohen, Mitchell B., MD, Bokulic, Ronald, MD, Deutsch, Gail, MD, Choudhary, Arabinda, MD, Falcone, Richard A., MD, Grabowski, Gregory A., MD

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Somatic HRAS p.G12S Mutation Causes Woolly Hair and Epidermal Nevi von Levinsohn, Jonathan L., Teng, Joyce, Craiglow, Brittany G., Loring, Erin C., Burrow, T Andrew, Mane, Shrikant S., Overton, John D., Lifton, Richard P., McNiff, Jennifer M., Lucky, Anne W., Choate, Keith A.

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Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1 von Mistry, Pramod K., Balwani, Manisha, Baris, Hagit N., Turkia, Hadhami Ben, Burrow, T. Andrew, Charrow, Joel, Cox, Gerald F., Danda, Sumita, Dragosky, Marta, Drelichman, Guillermo, El-Beshlawy, Amal, Fraga, Cristina, Freisens, Selena, Gaemers, Sebastiaan, Hadjiev, Evgueniy, Kishnani, Priya S., Lukina, Elena, Maison-Blanche, Pierre, Martins, Ana Maria, Pastores, Gregory, Petakov, Milan, Peterschmitt, M. Judith, Rosenbaum, Hanna, Rosenbloom, Barry, Underhill, Lisa H., Cox, Timothy M.

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Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease von Elstein, Deborah, Burrow, T Andrew, Charrow, Joel, Giraldo, Pilar, Mehta, Atul, Pastores, Gregory M, Lee, Hak-Myung, Mellgard, Björn, Zimran, Ari

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Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1 von Mistry, Pramod K., Balwani, Manisha, Baris, Hagit N., Turkia, Hadhami Ben, Burrow, T. Andrew, Charrow, Joel, Cox, Gerald F., Danda, Sumita, Dragosky, Marta, Drelichman, Guillermo, El-Beshlawy, Amal, Fraga, Cristina, Freisens, Selena, Gaemers, Sebastiaan, Hadjiev, Evgueniy, Kishnani, Priya S., Lukina, Elena, Maison-Blanche, Pierre, Martins, Ana Maria, Pastores, Gregory, Petakov, Milan, Peterschmitt, M. Judith, Rosenbaum, Hanna, Rosenbloom, Barry, Underhill, Lisa H., Cox, Timothy M.

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Widespread Vasculopathy in a Patient with Morquio A Syndrome von Powell, Adam W, Taylor, Michael D, Burrow, T Andrew, Hopkin, Robert J, Prada, Carlos E, Jefferies, John L

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Non‐lethal congenital hypotonia due to glycogen storage disease type IV von Burrow, T. Andrew, Hopkin, Robert J., Bove, Kevin E., Miles, Lili, Wong, Brenda L., Choudhary, Arabinda, Bali, Deeksha, Li, Sing Chung, Chen, Yuan‐Tsong

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O18: A path forward for patients with glycogen branching enzyme deficiency: Consensus on diagnosing and managing glycogen storage disease type IV von Koch, Rebecca, Soler-Alfonso, Claudia, Kiely, Bridget, Asai, Akihiro, Smith, Ariana, Bali, Deeksha, Kang, Peter, Landstrom, Andrew, Akman, H. Orhan, Burrow, T. Andrew, Orthmann-Murphy, Jennifer, Goldman, Deberah, Pendyal, Surekha, El-Gharbawy, Areeg, Austin, Stephanie, Case, Laura, Schiffmann, Raphael, Hirano, Michio, Kishnani, Priya

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Enzyme reconstitution/replacement therapy for lysosomal storage diseases von Burrow, T Andrew, Hopkin, Robert J, Leslie, Nancy D, Tinkle, Bradley T, Grabowski, Gregory A

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