Publications by authors named "I Guichard"
Article Synopsis
- Some myopathies can cause dropped head or bent spine syndrome (DH/BS), but its significance in inflammatory myopathies (IM) hasn't been thoroughly explored.
- A study compared 49 patients with DH/BS related to IM to 98 control IM patients, finding that those with DH/BS were older and had more severe symptoms, including upper limb weakness, dysphagia, and muscle atrophy.
- The results suggest that DH/BS serves as a marker for the severity of IM and is often linked to complications related to systemic sclerosis, highlighting its clinical importance.
Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are the most prevalent monogenic disorders worldwide. Trial HGB-205 ( NCT02151526 ) aimed at evaluating gene therapy by autologous CD34 cells transduced ex vivo with lentiviral vector BB305 that encodes the anti-sickling β-globin expressed in the erythroid lineage. HGB-205 is a phase 1/2, open-label, single-arm, non-randomized interventional study of 2-year duration at a single center, followed by observation in long-term follow-up studies LTF-303 ( NCT02633943 ) and LTF-307 ( NCT04628585 ) for TDT and SCD, respectively.
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