Sustained efficacy and tolerability in infants and young children with life-threatening hypophosphatasia treated with asfotase alfa

2014 ◽  
Author(s):  
Michael Whyte ◽  
Jill Simmons ◽  
Richard Lutz ◽  
Marc Vallee ◽  
Agustin Melian ◽  
...  
Keyword(s):  
Young Children ◽  
Asfotase Alfa ◽  
Sustained Efficacy ◽  
Life Threatening ◽  
Infants And Young Children

Improvement in bone manifestations and respiratory status in infants and young children with HPP treated with asfotase alfa: an update on the ENB-010-10 trial

2015 ◽  
Author(s):  
Christine Hofmann ◽  
Cheryl Rockman-Greenberg ◽  
Paul Harmatz ◽  
Scott Moseley ◽  
Tatjana Odrljin ◽  
...  
Keyword(s):  
Young Children ◽  
Asfotase Alfa ◽  
Respiratory Status ◽  
Infants And Young Children

scholarly journals Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial

2019 ◽  
Vol 7 (2) ◽  
pp. 93-105 ◽  
Author(s):  
Michael P Whyte ◽  
Jill H Simmons ◽  
Scott Moseley ◽  
Kenji P Fujita ◽  
Nicholas Bishop ◽  
...  
Keyword(s):  
Young Children ◽  
Phase 2 ◽  
Open Label ◽  
Asfotase Alfa ◽  
Open Label Phase ◽  
Extension Trial ◽  
Infants And Young Children

scholarly journals Diagnosis and management after life threatening events in infants and young children who received cardiopulmonary resuscitation.

BMJ ◽  
1993 ◽  
Vol 306 (6876) ◽  
pp. 489-492 ◽  
Author(s):  
M P Samuels ◽  
C F Poets ◽  
J P Noyes ◽  
H Hartmann ◽  
J Hewertson ◽  
...  
Keyword(s):  
Cardiopulmonary Resuscitation ◽  
Young Children ◽  
Diagnosis And Management ◽  
Life Threatening ◽  
Infants And Young Children

scholarly journals Clinical application experience of asfotase alfa for a young patient with childhood hypophosphatasia

2019 ◽  
Vol 22 (1) ◽  
pp. 24-29
Author(s):  
Nataliya Y. Kalinchenko ◽  
Olga O. Golounina ◽  
Tatiana A. Grebennikova ◽  
Galina A. Melnichenko ◽  
Anatoly N. Tiulpakov ◽  
...  
Keyword(s):  
Alkaline Phosphatase ◽  
Age Of Onset ◽  
Childhood Onset ◽  
Systemic Complications ◽  
Enzyme Replacement ◽  
Asfotase Alfa ◽  
Life Threatening ◽  
Defective Bone ◽  
Reduced Activity ◽  
Infants And Young Children

Hypophosphatasia (HPP) is a rare hereditary metabolic disease characterized by defective bone and dental mineralization, systemic complications that lead to disability of patients. HPP is classified into six forms according to the age of onset and severity of its clinical picture. The disease is caused by a reduced activity of the tissue nonspecific alkaline phosphatase (TNSALP) and elevated concentrations of pyrophosphates. Asfotase alfa is the only pathogenetic enzyme replacement therapy with recombinant human bone-targeted TNSALP approved for treatment of patients with perinatal, infantile and juvenile‐onset HPP. This treatment is associated with improved skeletal mineralization, respiratory function and overall survival in infants and young children with life-threatening hypophosphatasia. The world experience in application of recombinant alkaline phosphatase in adults is very limited. We present a clinical case that describes the first Russian experience in the use of asfotase alfa in an 18-year-old patient with late diagnosis of childhood-onset HPP.

scholarly journals Edukasi Dengan Metode Audiovisual Sebagai Upaya Peningkatan Pengetahuan Kader Mengenai Bahaya Tersedak Pada Bayi

2020 ◽  
Vol 1 (2) ◽  
pp. 96-101
Author(s):  
Noerma Shovie Rizqiea ◽  
Ratih Dwilestari Puji Utami
Keyword(s):  
Health Education ◽  
Young Children ◽  
Good Knowledge ◽  
Test Results ◽  
Emergency Situations ◽  
Measurement Results ◽  
Post Test ◽  
Life Threatening ◽  
Respiratory Emergencies ◽  
Infants And Young Children

Background: Children are uniquely vulnerable to a variety of emergency situations. These situations are often life-threatening if not treated quickly and effectively. Infants and young children are at higher risk for respiratory emergencies than adolescents or adults. Young children often lack coordination, which makes them prone to choking on food and small objects, which can also lead to cardiac arrest. The aim of this community service is that after receiving health education and demonstrations about the dangers of choking, it hopes that an increase in cadres' knowledge of the dangers of choking at the Posyandu for toddlers, Sadar Ibu I, Dusun Jetak. Methods: Respondents involved were 14 cadres of Posyandu Toddler Sadar Ibu I Dusun Jetak. Activities carried out online. The activity began with a pre-test, then continued with provisions health education using audio-visual video media, and ended with a post-test measurement. Results: The percentage of knowledge of cadres during the pre-test showed that 3 cadres (21%) had good knowledge, and the remaining 11 cadres (79%) had less knowledge. The post-test results showed that 10 cadres (71%) had good knowledge, and the remaining 4 cadres (29%) still had a bad knowledge. Conclusion: Increase Cadre's knowledge of the dangers of choking on babies.

Interleukin-13 associates with life-threatening rhinovirus infections in infants and young children

2018 ◽  
Vol 53 (6) ◽  
pp. 787-795 ◽  
Author(s):  
Mauricio T. Caballero ◽  
Diego R. Hijano ◽  
Patricio L. Acosta ◽  
Cecilia G. Mateu ◽  
Débora N. Marcone ◽  
...  
Keyword(s):  
Young Children ◽  
Interleukin 13 ◽  
Life Threatening ◽  
Infants And Young Children
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