Researchers at Karolinska Institutet have discovered the underlying cause of a hereditary muscle disease first characterised in a Swedish family in 1980. It proves to be the first identified disease ...

New avenues are now being opened for future treatment of Laing distal myopathy, a rare disorder that causes muscles in the feet, hands and elsewhere to atrophy. In a study published in the journal ...

New avenues are now being opened for future treatment of Laing distal myopathy, a rare disorder that causes muscles in the feet, hands and elsewhere to atrophy. In a study published in the journal ...

A drug to treat distal myopathy — a rare disorder characterized by progressive weakness of the distal muscles of the hands and feet, with patients losing the ability to walk in roughly 10 years — was ...

GAITHERSBURG, Md.--(BUSINESS WIRE)--Leadiant Biosciences, Inc. today announced it has entered into a license agreement and cooperative research and development agreement (CRADA) with the National ...

NOVATO, Calif., Aug. 15, 2011 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced the ...

Eight patients (Table 1, 1.1–6) from six unrelated families were included in this study. The first 7 patients (1.1–5) were previously reported as suspected DMRV without genetic confirmation in Chinese ...

Medically reviewed by Brigid Dwyer, MD Key Takeaways Life expectancy for muscular dystrophy depends on the type and severity ...

I am studying Laing Distal Myopathy (MPD1), an inherited condition that causes progressive muscle weakness in the extremities and has no cure. MPD1 is caused by mutations in β-myosin, the primary ...

Mean change in UEC total score from the start of the Phase2/3 study (Week 0) to the final evaluation time point (Week 120) of the extension study. Closed circle and solid line, SA-ER/SA-ER group; open ...

Initially, the team attributed the muscle weakness to either HIV or drug-mediated myopathy but a muscle biopsy later revealed atrophy of type II fibers without necrosis or inflammation. A case ...