Researchers developed a nanopore sequencing pipeline that may improve genetic diagnosis of repeat expansion disorders, ...
The oral combination of azacitidine and cedazuridine (ASTX030) met its primary endpoint in phase 3 AZTOUND study of patients ...
A woman with chronic inflammatory demyelinating polyneuropathy (CIDP) shares what it was like to walk in public for the first ...
Shortness of breath seems to be worse in patients with IPF, while cough and quality of life seem worse in patients with PPF.
Older ambulatory patients with DMD who received delandistrogene moxeparvovec in clinical trials maintained more motor ...
Allogeneic hematopoietic cell transplantation achieved a 3-year overall survival of 69% in adults aged 18 to 40 years with ...
Shear wave elastography and ultrasound-derived indices exhibit excellent diagnostic performance in detecting interstitial ...
Although the phase 2b study of SOM3355 for HD-associated chorea missed its primary endpoint, participants not taking ...
Plasma protein biomarkers of epithelial injury and fibrogenesis may be associated with short-term disease progression in ...
Researchers adapted the MDS-NMS tool to assess nonmotor symptoms in Huntington disease (HD), with preliminary results ...
A mother of 2 sons with FSGS explains why routine lab results can be intimidating and how patient education could help change ...
In a Dravet syndrome (DS) mouse study, hippocampal NaV1.1 restoration reduced seizure frequency; a multisite approach also ...
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