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After more than a decade without seeing a neurologist for my limb-girdle muscular dystrophy (LGMD), I was finally able to get connected with a specialist after attending my very first Muscular ...

The discontinuation was not driven by safety concerns, as emugrobart was well tolerated, with no serious adverse events resulting in study withdrawal. Roche and Genentech have announced that they are ...

Physiotherapy is the best way to keep children with DMD on their feet as long as possible, but my grandson is just a boy who wants to play. A New Genetic Test Clarified My Muscular Dystrophy Diagnosis ...

Physicians should actively involve caregivers in treatment decisions and provide clear expectations regarding treatment efficacy and adverse effects. An international panel of experts provided ...

Patients with PBC treated with linerixibat had significant, rapid, and sustained improvements in cholestatic pruritus and sleep interference related to pruritus compared with the placebo. The US Food ...

Among those with ATTR, the sodium-glucose cotransporter 2 inhibitor group saw a nearly 50% reduction in all-cause mortality compared with controls (9.43% vs 19.2%; HR, 0.52). Sodium-glucose ...

Overall survival was similar between groups, likely due to effective BTK inhibitor salvage after lenalidomide relapse. Bruton tyrosine kinase inhibitor (BTKi) maintenance therapy was associated with ...

Lower rates of chronic and acute GVHD, infections, and nonrelapse mortality indicate a substantially reduced toxicity burden. In patients with myelodysplastic syndrome (MDS) and acute leukemias ...

(Getty Images) The analysis included 486 HELIOS-B participants with KCCQ assessments at both baseline and 30 months. The greatest differences in score change occurred in questionnaire items gauging ...

Around a third (33.6%) of patients with cirrhosis were tested for alpha-1 antitrypsin deficiency (AATD). The rates of testing for alpha-1 antitrypsin deficiency (AATD) are low among veterans with ...

IgG MGUS was associated with less frequent acute onset (6% vs 33%) and more frequent sensory deficits at onset (94% vs 67%). Immunoglobulin G (IgG) monoclonal gammopathy of undetermined significance ...