9 research outputs found
Computational Controversy
Climate change, vaccination, abortion, Trump: Many topics are surrounded by
fierce controversies. The nature of such heated debates and their elements have
been studied extensively in the social science literature. More recently,
various computational approaches to controversy analysis have appeared, using
new data sources such as Wikipedia, which help us now better understand these
phenomena. However, compared to what social sciences have discovered about such
debates, the existing computational approaches mostly focus on just a few of
the many important aspects around the concept of controversies. In order to
link the two strands, we provide and evaluate here a controversy model that is
both, rooted in the findings of the social science literature and at the same
time strongly linked to computational methods. We show how this model can lead
to computational controversy analytics that have full coverage over all the
crucial aspects that make up a controversy.Comment: In Proceedings of the 9th International Conference on Social
Informatics (SocInfo) 201
Long-term observational, non-randomized study of enzyme replacement therapy in Late-Onset Glycogenosis type II
Objectives Type II glycogenosis (GSDII) is a lysosomal storage disorder due to acid alpha-glucosidase (GAA) deficiency. Enzyme replacement therapy (ERT) with human recombinant alpha-glucosidase (rhGAA) has been demonstrated to be effective in the treatment of infantile forms of GSDII, but little information is available concerning late-onset phenotypes. Long-term follow-up studies are not available at present. The aim of this study was to evaluate the ERT long-term effects in late-onset GSDII.
Methods Twenty-four patients, including 7 juveniles and 17 adults, received bi-weekly infusion of rhGAA (20 mg/kg) for at least 36 months. Clinical conditions, muscular function (6-min walking test, 6MWT; Walton scale, WS), respiratory function (vital capacity, VC; forced expiratory volume, FEV1; arterial pCO(2)), and muscle enzymes were assessed every 6 months.
Results The 6MWT improved in both juvenile and adult patients (p = 0.01, p = 0.0002, respectively), as well as in patients with moderate to severe muscle function impairment (WS > 3.5; p = 0.002). An overall improvement in WS was also observed (p = 0.0003). VC and FEV1 remained unchanged, while pCO(2) decreased (p = 0.017). Muscle enzymes decreased significantly (p < 0.0001). Two patients (8%) showed transient secondary events during ERT.
Conclusions Long-term ERT with rhGAA was shown to be safe, well tolerated, and effective in improving motor function and in stabilizing respiratory function in late-onset GSDII. The response pattern showed a progressive clinical improvement during the follow-up period in juvenile patients, while in adults it reached and maintained a plateau after the first year of treatment
Long-term observational, non-randomized study of enzyme replacement therapy in late-onset glycogenosis type II.
Objectives Type II glycogenosis (GSDII) is a lysosomal
storage disorder due to acid alpha-glucosidase (GAA)
deficiency. Enzyme replacement therapy (ERT) with
human recombinant alpha-glucosidase (rhGAA) has been
demonstrated to be effective in the treatment of infantile
forms of GSDII, but little information is available
concerning late-onset phenotypes. Long-term follow-up
studies are not available at present. The aim of this study
was to evaluate the ERT long-term effects in late-onset
GSDII.
Methods Twenty-four patients, including 7 juveniles and 17
adults, received bi-weekly infusion of rhGAA (20 mg/kg)
for at least 36 months. Clinical conditions, muscular
function (6-min walking test, 6MWT; Walton scale, WS),
respiratory function (vital capacity, VC; forced expiratory
volume, FEV1; arterial pCO2), and muscle enzymes were
assessed every 6 months.
Results The 6MWT improved in both juvenile and adult
patients (p=0.01, p=0.0002, respectively), as well as in
patients with moderate to severe muscle function impairment
(WS>3.5; p=0.002). An overall improvement in WS
was also observed (p=0.0003). VC and FEV1 remained
unchanged, while pCO2 decreased (p=0.017). Muscle
enzymes decreased significantly (p<0.0001). Two patients
(8%) showed transient secondary events during ERT.
Conclusions Long-term ERT with rhGAA was shown to
be safe, well tolerated, and effective in improving motor
function and in stabilizing respiratory function in lateonset
GSDII. The response pattern showed a progressive clinical improvement during the follow-up period in
juvenile patients, while in adults it reached and maintained
a plateau after the first year of treatment