Cardiac scintigraphy had higher sensitivity and specificity across different radiotracers in diagnosing cardiac amyloidosis compared to cardiac magnetic resonance imaging. Cardiac scintigraphy, ...
An ongoing open-label extension study suggests that the therapy's benefits may extend through at least 52 weeks. Deupirfenidone (LYT-100), a next-generation antifibrotic therapy, demonstrated a ...
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare acquired autoimmune neurological disorder in which both T-cell-mediated and humoral immune mechanisms target healthy myelinated ...
Sickle cell disease is inherited in an autosomal recessive pattern, which means that a child is born with SCD only when they inherit 2 defective copies of the sickle cell gene (1 from each parent). If ...
In CIDP, SOC therapies (immunoglobulins/corticosteroids) are burdensome, have variable efficacy, and significant side-effects. Two phase 3 clinical trials, MOBILIZE ...
Nusinersen, an SMN2-targeted antisense oligonucleotide drug administered by intrathecal injection, was able to improve motor function and survival. Treatment with nusinersen can improve motor function ...
Between 2002 and 2020, the incidence and prevalence of AATD increased significantly. At the end of 2020, the incidence and prevalence of alpha-1 antitrypsin deficiency (AATD) in Sweden were 1.83 and ...
Obeticholic acid, seladelpar, and elafibranor all significantly increased the biochemical response rate. Elafibranor seems to be slightly more effective than seladelpar as a second-line therapy in ...
Individuals with advanced liver disease may also experience significant weight loss due to nausea and poor appetite. Candidates for liver transplantation may need to gain weight to optimize surgical ...
The Stride Velocity 95th Centile (SV95C) showed sensitivity to a decline in the ability to walk over short intervals in patients with Duchenne muscular dystrophy (DMD), according to a new study ...
This unique case underscores the importance of meticulous prenatal and postnatal immunologic workup when alloimmune conditions are suspected. A 37-year-old multiparous woman with RhD alloimmunization ...
The researchers used the Paris criteria to reach a final diagnosis. A team of international researchers presented the rare case of a male patient with autoimmune hepatitis-primary biliary cholangitis ...
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