6 research outputs found
Orthopedic manifestations in patients with mucopolysaccharidosis type II (Hunter syndrome) enrolled in the Hunter Outcome Survey
- Author
- Publication venue
- PAGEPress Publications
- Publication date
- 01/07/2010
- Field of study
Mucopolysaccharidosis type II (MPS II or Hunter syndrome) is a rare, inherited disorder caused by deficiency of the lysosomal enzyme iduronate-2-sulfatase. As a result of this deficiency, glycosaminoglycans accumulate in lysosomes in many tissues, leading to progressive multisystemic disease. The cardiopulmonary and neurological problems associated with MPS II have received considerable attention. Orthopedic manifestations are common but not as well characterized. This study aimed to characterize the prevalence and severity of orthopedic manifestations of MPS II and to determine the relationship of these signs and symptoms with cardiovascular, pulmonary and central nervous system involvement
Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II
- Publication venue
- 'Wiley'
- Publication date
- 01/04/2008
- Field of study
Enzyme Replacement Therapy with Imiglucerase in a Taiwanese Child with Type 1 Gaucher Disease
- Publication venue
- 'Elsevier BV'
- Publication date
- 01/05/2006
- Field of study
The treatment of type 1 Gaucher disease has dramatically improved with the development of enzyme replacement therapy (ERT). To date, however, imiglucerase treatment of this disease in Taiwanese pediatric patients has not been reported. A Taiwanese child with type 1 Gaucher disease was regularly treated with imiglucerase beginning October 1998. This 12-year 10-month-old boy had undergone splenectomy when he was 4 years old. He received intravenous imiglucerase 60 U/kg every 2 weeks for 78 months. No signs of pubertal development were documented at the commencement of ERT. There were no serious adverse effects. The patient had significant improvement in skeletal deformity, a dramatic decrease in liver size, markedly increased linear growth, alleviation of bone pain and bone crises, correction of anemia, and improved bone mineral density. ERT with imiglucerase improved the quality of life in this child with type 1 Gaucher disease
Parental social support, coping strategies, resilience factors, stress, anxiety and depression levels in parents of children with MPS III (Sanfilippo syndrome) or children with intellectual disabilities (ID)
- Author
- Publication venue
- 'Springer Science and Business Media LLC'
- Publication date
- 01/01/2012
- Field of study
in the Hunter Outcome Survey
- Author
- Publication venue
- Publication date
- Field of study
in patients with muco-polysaccharidosis type II (Hunter syndrome) enrolle
Drug monographs
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- Omari
- Omenaca
- Omeñaca
- Oncel
- Onland
- Onwuneme
- Orenstein
- Orme
- Ortega
- Osadchy
- Osborn
- Osendarp
- Ozdemir
- Ozkiraz
- Ozturk
- Pabuccu
- Pacifici
- Pacifici
- Packman
- Pacqué
- Padari
- Page
- Pagel
- Pai
- Paidas
- Paidas
- Painter
- Painter
- Palladino
- Palma
- Palmer
- Palmer
- Palmieri
- Palombi
- Pan
- Panburana
- Papatsonis
- Papoff
- Pappas
- Pappert
- Paradisis
- Paradisis
- Paradisis
- Parisi
- Parke
- Parr
- Pasley
- Pasternak
- Pasternak
- Patel
- Patel
- Patole
- Paul
- Paul
- Payne
- Pearce
- Pearson
- Pellicer
- Peltola
- Pennell
- Perlman
- Perondi
- Perry
- Perry
- Pershad
- Pessoa-Silva
- Peter
- Petersen
- Petersen
- Petersen
- Phelps
- Philippi
- Phillips
- Phrommintikul
- Piastra
- Piatt
- Pichichero
- Pierce
- Pijpers
- Pilgrim
- Pillekamp
- Pilwoz
- Piotrowski
- Piper
- Pitman
- Pitt
- Piña-Garza
- Plan
- Plaud
- Plaud
- Playfor
- Playfor
- Plowe
- Poets
- Pohlandt
- Poindexter
- Pokela
- Pokela
- Polak
- Polin
- Polin
- Pollock
- Polycarpou
- Porcelijn
- Porcelli
- Porta
- Poterjoy
- Pourcyrous
- Powell
- Prager
- Prasad
- Pratap
- Presti
- Price
- Pritchard
- Prober
- Pruthi
- Pryce
- Prymula
- Puliyanda
- Pullen
- Pullen
- Pun
- Purcell
- PĂ©rez-Ayuso
- PĂ©zarda
- Quarteman
- Quine
- Quinzii
- Radde
- Rademaker
- Radicioni
- Radloff
- Rahman
- Rahman
- Rahman
- Rahman
- Rains
- Rajapakse
- Raju
- Raju
- Ramaekers
- Ramaiah
- Ramantani
- Ramasethu
- Ramusovic
- Rand
- Randolph
- Rank
- Rankin
- Rao
- Rapp
- Rapp
- Rashid
- Raszka
- Ratcliff
- Ratjen
- Rautakorpi
- Ray
- Reading
- Rector
- Reed
- Reed
- Reed
- Reeves
- Remington
- Remington
- Renfrew
- Resch
- Resino
- Rey
- Reyes
- Reynolds
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- Richards
- Rieder
- Rigden
- Rijken
- Rimensberger
- Risson
- Rivera
- Robbins
- Robel-Tillig
- Roberge
- Roberts
- Roberts
- Roberts
- Roberts
- Roberts
- Robertson
- Robinson
- Rode
- Roehr
- Roifman
- Rojchgot
- Rolfe
- Romagnoli
- Romero-Cabello
- Ronge
- Roos
- Rosa
- Roschitz
- Rose
- Rose
- Rosen
- Rosenblatt
- Rosenfeld
- Rosenfeld
- Rossignol
- Rossignol
- Rossignol
- Roth
- Rothman
- Rouse
- Rouse
- Rowe
- Roy
- Royal College of Obstetricians and Gynaecologists
- Rozance
- Russell
- Russo
- Rutter
- Ryan
- Rötig
- Saarenmaa
- Saarenmaa
- Saez-Lorens
- Sagirli
- Saha
- Saiman
- Salbert
- Saleh
- Salim
- Salomon
- Salsky
- Salvatore
- Salviati
- Sanchez-Ramos
- Saneto
- Sankar
- Sans
- Santiago
- Sarkar
- Sato
- Sauberan
- Saugstad
- Saul
- Savino
- Sazawal
- Scaglia
- Schaaf
- Schaaf
- Schanler
- Schaschini
- Scheiber
- Scher
- Schiff
- Schlagenhauf
- Schlagenhauf
- Schmidt
- Schmidt
- Schmidt
- Schmidt
- Schmidt
- Schoeffler
- Schortgen
- Schrag
- Schramm
- Schrum
- Schuchat
- Schuh
- Schuler
- Schulze-Neick
- Schwarze
- Scott
- SEAQUAMAT Trial Group
- Segar
- Seguin
- Seikaly
- Seri
- Sexson
- Shah
- Shah
- Shah
- Shah
- Shah
- Shah
- Shah
- Shama
- ShangGuan
- Shannon
- Shany
- Shany
- Shapiro
- Sharpe
- Sheele
- Shekerdemain
- Shellhaas
- Shenai
- Shennan
- Shepard
- Sher
- Sheridan
- Sherlock
- Sherwin
- Shetty
- Shi
- Shiball
- Shierhout
- Shih
- Shim
- Shingadia
- Shinjoh
- Shoemaker
- Shorten
- Shrimpton
- Sicherer
- Silvers
- Simbi
- Simcox
- Simon
- Simons
- Simonse
- Sims
- Singh
- Singh
- Singh
- Sinha
- Sistonen
- Skevaki
- Skinner
- Skinner
- Skopnick
- Slater
- Slaughter
- Slim
- Smederley
- Smedsaas-Löfvenberg
- Smellie
- Smith
- Smith
- Smith
- Smith
- Smith
- Smith
- Smits-Wintjens
- Smoak
- Smyth
- So
- Soar
- Sobel
- Soditt
- Sohn
- Sola
- Solomon
- Soltani
- Song
- Sonnappa
- Sood
- Sorbie
- Sorensen
- Soukka
- Souza
- Sowunmi
- Soyer
- Soysal
- Spanorfer
- Sparr
- Sparr
- Speer
- Spyridis
- Sreenan
- Sreeram
- Srinivasan
- Srinivasan
- Srinivasan
- Stacpoole
- Stacpoole
- Stacpoole
- Staedke
- Stafstrom
- Stalker
- Stalldecker
- Standing
- Stanek
- Stapleton
- Stathopoulos
- Steadman
- Steele
- Steer
- Steer
- Steinberg
- Steinhorn
- Stenson
- Stephens
- Stephens
- Stevens
- Stocker
- Stothard
- Stothard
- Strehle
- Streif
- Streif
- Strich
- Stuart
- Su
- Su
- Su
- Subhani
- Sullins
- Sullivan
- Sullivan
- SUMMIT Trial Study Group
- Suomalainen
- Suormala
- SUPPORT Study Group of the Eunice Kennedy Shriver NICHD Neonatal Research Network
- Surridge
- Sury
- Suyagh
- Suyagh
- Swan
- Sweet
- Sweet
- Sweetman
- SYROCOT (Systematic Review on Congenital Toxoplasmosis) study group
- SYROCOT (Systematic Review on Congenital Toxoplasmosis) study group
- Szajewska
- Szilagi
- SĂĄez-Llorens
- SĂžrensen
- Tack
- Taddio
- Taddio
- Tagbor
- Tagbor
- Taha
- Takeoka
- Tammachote
- Tamura
- Tan
- Tan
- Tanaka
- Tang
- Tanney
- Tasker
- Tata
- Taylor
- Taylor
- Taylor
- Taylor
- Taylor
- Taylor
- Taylor
- Taylor
- Taylor
- Taylor
- te Pas
- te Raa
- Tebruegge
- Tekgunduz
- Tenenbein
- Teo
- Terragna
- Tessin
- Tetzlaff
- Thayyil-Sudhan
- The Special Feed Working Group of the Paediatric Group of the British Dietetic Association
- Theilen
- Theilen
- Theis
- Thingvoll
- Thomas
- Thomas
- Thomas
- Thomas
- Thomas
- Thompson
- Thompson
- Thuy
- Tielsch
- Tiffany
- Tighe
- Tighe
- Timsit
- Tinnion
- Tirotta
- Tobin
- Tod
- Tolia
- Tolsa
- Tomsits
- Tookey
- Tookey
- Tookey
- Tookey
- Toppare
- Tourneux
- Tourneux
- Tran
- Trang
- Tremoulet
- Tripathi
- Trudel
- Trunz
- Tsatsaris
- Tsatsaris
- Tse
- Tsilou
- Tsui
- Tubman
- Tulloch
- Tulloch
- Tulloch
- Tullu
- Turnidge
- Turrentine
- Tyler
- Tylki-Szymanska
- TĂ€htinen
- Ugwamadu
- UK Group on Transmitted HIV Drug Resistance
- Urien
- Uslu
- Valentine
- Valenzuela
- Valerio
- Vallejo
- Vamhauwere
- Van de Velde
- van den Akker
- van den Anker
- van den Anker
- van den Anker
- van den Broek
- van den Broek
- van den Broek
- van den Oever
- van der Burg
- van der Heide-Jalving
- Van der Poll
- van der Torn
- van Donselaar-van der Pant
- van Enk
- Van Hasselt
- van Lingen
- Van Neil
- Van Overmeire
- van Rheenen
- van Rooij
- van Rooij
- Van Rooij
- van Vonderen
- Vanclooster
- Vandenplas
- Vandenplas
- Vandenplas
- Vanhatalo
- Vanier
- Vanstone
- Vargas
- Vargas-Origel
- Varner
- Vauzelle-Kervroëdan
- Veldman
- Veldman
- Vellodi
- Venkatesh
- Venkatesh
- Venkatesh
- Venkatesh
- Venter
- Vermes
- Vesikari
- Vestergaard
- Veuvonen
- Victorin
- Villain
- Villanueva-GarcĂa
- Vincent
- Viscardi
- Viscardi
- Vitali
- von Kries
- von Lindern
- von Rosensteil
- VĂĄsquez-Mendoza
- Wade
- Wadsworth
- Wagner
- Wainer
- Waites
- Walker
- Wallon
- Wan
- Wang
- Wang
- Wapner
- Ward
- Ward
- Wardle
- Wardle
- Wardle
- Wastell
- Watanabe
- Wathen
- Watkins
- Watkins
- Watkins
- Watt
- Weale
- Weber
- Weber
- Webster
- Weitkamp
- Wells
- Welzing
- Welzing
- Welzing
- Wenzl
- Werlin
- Werner
- West
- Westergren
- Wheless
- White
- White
- Whitelaw
- Whitelaw
- Whitelaw
- Whitfield
- Whitington
- Whitney
- Wiedmeier
- Wiholm
- Wijburg
- Wijburg
- Wijburg
- Wilby
- Wilcken
- Wilken
- Wilkes
- Wilkins
- Williams
- Williams
- Williams
- Williams
- Williams
- Willmore
- Willot
- Wills
- Wilmer
- Wilson
- Wilson
- Wirth
- Withington
- Wolf
- Wolf
- Wolf
- Wolfe
- Wong
- Woo
- Woodrow
- Worldwide Atosiban versus Beta-agonists Study Group
- Wraith
- Wraith
- Wren
- Wright
- Wright
- Wu
- Wurthwein
- Wyckoff
- Wyckoff
- Wyllie
- Wyllie
- Wynn
- Xie
- Xinias
- Yalaz
- Yanni
- Yaseen
- Yates
- Yeh
- Yeh
- Yeung
- Yoshida
- Young
- Young
- Young
- Young
- Yukawa
- YĂŒksel
- Zakrzewska-Bode
- Zaman
- Zanetti
- Zar
- Zeidan
- Zelicof-Paul
- Zelnik
- Zempleni
- Zeng
- Zhanel
- Zhang
- Zhao
- Ziaei
- Zuppa
- Publication venue
- 'Wiley'
- Publication date
- Field of study