Rare Disease Advisor, a trusted source of medical news and feature content for healthcare providers, offers clinicians insight into the latest research to inform clinical practice and improve patient ...
Upper-to-lower body segment ratio decreased significantly from baseline relative to untreated comparators in both age groups. Continuous treatment with vosoritide in children with achondroplasia shows ...
A decision tree integrating left ventricular ejection fraction, global longitudinal strain, and cardiac index identified 4 prognostic groups with hazard ratios for 4-year mortality ranging between 1.6 ...
Chronic inflammatory demyelinating polyneuropathy (CIDP) is an immune-mediated neuromuscular disorder affecting the peripheral nervous system. CIDP is characterized by gradual sensory loss and muscle ...
Test Your Knowledge About the Genetics of Chronic Lymphocytic Leukemia Test Your Knowledge About the Prognosis of Chronic Lymphocytic Leukemia Test Your Knowledge About the Diagnosis of Chronic ...
Kine Sciences advanced KINE-101 to the phase 1b/2a study following the successful completion of a phase 1 study in the United States. The first patient in a new phase 1b/2a clinical trial to ...
Baseline hemoglobin at diagnosis and patient age independently predict transfusion density, whereas other hematologic factors do not. A higher cumulative red blood cell (RBC) transfusion burden is ...
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 ...
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 ...
The whole body production, plasma concentrations, and intra- and extracellular compartmental analyses of various amino acids were attenuated in DMD dogs given beta-hydroxy-beta-methylbutyrate (HMB).
Belinda Moser of ExamOne takes a blood sample from Marie Bedirlian, whose 14-year-old son, Brayden, has Duchenne. Bedirlian is a DMD carrier. (Photo by Larry Luxner) Pradeep Mammen, MD, an expert on ...
In a nonhuman primate model of acquired hemophilia A, treatment with PS-siRNA 40031 restored thrombin generation to normal levels. Reducing protein S (PS) levels can increase thrombin generation and ...
Results that may be inaccessible to you are currently showing.
Hide inaccessible results