A study to assess the efficacy and safety of KRN23 in adults with X-linked Hypophosphatemia (XLH) Establish the effect of KRN23 treatment compared with placebo on increasing serum phosphorus levels in adults with XLH

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A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) Estudio para evaluar la seguridad y la eficacia de UX007 en pacientes con trastornos del movimiento asociados al síndrome de deficiencia del transportador de glucosa tipo 1 (Glut1 DS) Primary Objectives: •Evaluate the efficacy and safety of UX007 in the treatment of disabling paroxysmal movement disorders associated with Glut1 DS El objetivo principal de este estudio es: •Eva...

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A study to see if sialic acid is safe and effective in treating people with Hereditary Inclusion Body Myopathy (HIBM), a rare muscle disease Evaluate the effect of 6 g/day SA-ER treatment of subjects with GNEM on upper extremity muscle strength (UEC score) as measured by dynamometry

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