Search Results - "Stolwijk, Nina N."

Access to medicines for rare diseases: A European regulatory roadmap for academia by Rosenberg, Noa, van den Berg, Sibren, Stolwijk, Nina N, Jacobs, Bart A W, Post, Hendrika C, Pasmooij, Anna M G, de Visser, Saco J, Hollak, Carla E M

“…: Novel or repurposed medicines for rare diseases often emerge from fundamental research or empirical findings in academia. However, researchers may be…”
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Recurrent metabolic alkalosis following ketone body treatment of adult mitochondrial trifunctional protein deficiency: A case report by Stolwijk, Nina N., Langeveld, Mirjam, Jacobs, Bart A. W., Vogt, Liffert, Haverkamp, Jorien A., Ferdinandusse, Sacha, Hollak, Carla E. M.

“…Recent studies have reported the potential for the therapeutic use of ketones in the form of ketone salts (KSs) in pediatric patients with fatty acid oxidation…”
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Development of medicines for rare diseases and inborn errors of metabolism: Toward novel public-private partnerships by Rosenberg, Noa, Stolwijk, Nina N, van den Berg, Sibren, Heus, Joris J, van der Wel, Vincent, van Gelder, Teun, Bosch, Annet M, de Visser, Saco J, Hollak, Carla E M

“…Medicine development for rare diseases, including inborn errors of metabolism (IEMs) is challenging. Many academic innovations fail to reach the patient,…”
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Repurposing empagliflozin in individuals with glycogen storage disease Ib: A value‐based healthcare approach and systematic benefit‐risk assessment by Derks, Terry G. J., Venema, Annieke, Köller, Clara, Bos, Eline, Overduin, Ruben J., Stolwijk, Nina N., Hofbauer, Peter, Bolhuis, Mathieu S., Eenennaam, Fred, Groen, Henk, Hollak, Carla E. M., Wortmann, Saskia B.

“…Off‐label repurposing of empagliflozin allows pathomechanism‐based treatment of neutropenia/neutrophil‐dysfunction in glycogen storage disease type Ib (GSDIb)…”
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Morbidity of a Single Incision Transvaginal Mesh to Correct Apical Prolapse by Jeffery, Stephen T., Kortz, Brita S., Muavha, Dakalo, Stolwijk, Nina N., Ras, Lamees, Roovers, Jan-Paul W.R.

“…To determine complications and related reintervention rates associated with use of the Uphold Vaginal Support System (Boston Scientific, Boston, MA) for…”
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