Results 101 to 110 of about 32,062 (289)

Real‐World Outcomes of Prophylaxis With rIX‐FP in Germany: A Prospective, Non‐Interventional Study in Haemophilia B

Haemophilia, EarlyView.
ABSTRACT Introduction Efficacy and safety of albutrepenonacog alfa (rIX‐FP) in people with haemophilia B (PwHB) has been previously demonstrated in clinical trials. However, real‐world data insights are required. Aim To describe real‐world effectiveness and tolerability of rIX‐FP in PwHB in Germany.
Johannes Oldenburg, Swee Wenning, Susanne Holzhauer, Wolfgang Miesbach, Robert Klamroth, Thomas Linhoff, Patrick Sommerer, Christian Pfrepper, Martin Olivieri, all study investigators   +9 more
wiley   +1 more source

HAEMATURIA IN PATIENTS WITH HAEMOPHILIA

Zdravniški Vestnik, 2008
BACKGROUND A review article intends to discover a cause, frequency, treatment and complications ofhaematuria in patients with haemophilia. METHODS A review of PubMed (1969–2007) for key words haemophilia, haematuria and renaldisease. RESULTS Four studies
Majda Benedik Dolničar
doaj  

CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice

Scientific Reports, 2017
Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology.
Tsukasa Ohmori, Yasumitsu Nagao, Hiroaki Mizukami, Asuka Sakata, Shin-ichi Muramatsu, Keiya Ozawa, Shin-ichi Tominaga, Yutaka Hanazono, Satoshi Nishimura, Osamu Nureki, Yoichi Sakata   +10 more
doaj   +1 more source

Paediatric care of the child with haemophilia. [Elektronisk resurs]

, 2002
The paediatric care of children with haemophilia in developed countries should focus on the health of the child, not on the disorder. Gene therapy offers the hope of an ultimate 'cure' for the disorder, but until this is a viable proposition, patients ...
Ljung, Rolf,, Lund University.
core   +1 more source

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source

Rare Bleeding Disorders

, 2010
The best known and most common bleeding disorders are Haemophilia A (Factor VIII deficiency), Haemophilia B (Factor IX deficiency) and von Willebrand Disease. However, these do not represent all bleeding disorders.
Irish Haemophilia Society (IHS)
core  

Postural adjustment after an unexpected perturbation in children with haemophilia

, 2012
. Children with haemophilia often bleed inside joints and muscles, which may impair postural adjustments. These postural adjustments are necessary to control postural balance during daily activities.
Tanaka, C., De Souza, F. M. B., Villaca, P., Do Carmo, C. M., De Mello, M. H. M., Minuque, N. P., Pereira, R. P.   +6 more
core   +1 more source

Lack of Inhibitor Development in Previously Treated People With Haemophilia Switching Factor Products—Final Results of Athn 2: Factor Switching Study

Haemophilia, EarlyView.
Erin Cockrell, Martin Chandler, Tammuella Chrisentery‐Singleton, Stacy E. Croteau, Nabil Daoud, Michael F. Guerrera, M. Nikki Hirsh, Janna Journeycake, Roshni Kulkarni, Susan U. Lattimore, Catherine McGuinn, Margaret Ragni, Michael Recht, Ulrike M. Reiss, Robert Sidonio Jr., Cliffford M. Takemoto, Michael Tarantino, Annette von Drygalski, Ellis J. Neufeld   +18 more
wiley   +1 more source

Use of an Oral Health‐Related Quality of Life Instrument to Measure Unmet Dental Care Needs in Adults With Inherited Bleeding Disorders

Haemophilia, EarlyView.
ABSTRACT Introduction Preventive dental care is vital for individuals with bleeding disorders to reduce the need for potentially invasive procedures. Although dental care is a mandated function of U.S. federally supported hemophilia treatment centers (HTCs), access to dental care is widely variable.
Miguel A. Escobar, Megan M. Ullman, Joanna Larson, Michael M. Chan, Marisela Trujillo   +4 more
wiley   +1 more source

Physical Activity and Sedentary Behaviour in Children With Haemophilia

Haemophilia, EarlyView.
ABSTRACT Introduction Physical activity (PA) is vital in child development, while inactivity remains a leading contributor to global mortality. Therapeutic advances have enabled greater PA in boys with haemophilia (BwH), yet robust UK data on objectively measured PA and sedentary behaviour (SB) across severities are limited.
Melanie Bladen, Lucy Alderson, Eleanor Main, Mario Cortina‐Borja   +3 more
wiley   +1 more source