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Accurate diagnosis of patients with Morquio A can be challenging, particularly for individuals with more slowly progressing disease, which makes confirmation of disease diagnosis by molecular analysis valuable.
However, since patients with NTDT usually have a milder and more slowly progressing phenotype than β-thalassemia major patients have, there is a risk that regular monitoring and treatment may be delayed until complications become obvious.
However, there is a subset of female Fabry patients whose disease symptoms are relatively mild or more slowly progressing who currently do not receive ERT.
Patients with high Gleason scores, poor response to initial ADT, rapidly progressive disease, or poorly controlled symptoms may derive greater benefit from immediate chemotherapy, while a trial of abiraterone may be reasonable in patients with less extensive or more slowly progressing disease.
The occlusion can be achieved by introducing a cathetermediated embolization coil or a surgical ligature to block the coronary flow, or by surgically placing ameroid constrictors around the coronary vessels to resemble more slowly progressing occlusion (Watanabe et al., 1998; Hughes et al., 2003; Madeddu et al., 2006; Abarbanell et al., 2010; Gao et al., 2010) (Table 1).
For these purposes, surgeries were tailored to produce less joint instability with the intent of producing a more slowly progressing disease than the standard anterior cruciate ligament (ACL) models as has been recently described for the DMM and other models [ 31, 34].
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West African trypanosomiasis, which circulates in the human environment, is characterised by a more benign, slowly progressing and chronic clinical course of the disease.
However, clinical disease manifestation can also progress more slowly with mild anaemia, progressive renal insufficiency and oscillating thrombocytopaenia [ 7].
The development of animal models is progressing more slowly.
Partnerships with industry are driving rapid translation of this research into human trials, while basic research is progressing more slowly.
But those efforts appear to be progressing more slowly, in part because there is less of a vacuum to fill.
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