1 Institute of Genetic Medicine, International Centre for Life, Newcastle University, Newcastle upon Tyne, UK 2 Department of Neurology, Royal Victoria Infirmary, Newcastle upon Tyne, UK Methods We ...

1 Department of Clinical Development, Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan 2 Department of Neuromuscular Research, National Institute of Neuroscience ...

CIM and CIP are common complications of critical care. Both CIM and CIP cause limb weakness and weakness of muscles used for breathing. CIM and CIP make people more unwell, increase mortality and slow ...

The sources on MONDO:0018773 indicate HSPB8 as the causative gene, same as https://omim.org/entry/621078 on MONDO:0976133. And OMIM 621078 and Orpha 476093 https ...

1 Man, 3 Hearts: Bergen County Survivor Whose Story Touched Thousands Dies At 41 Christophe Lafontant, a New Jersey man who inspired thousands through his fight against rare disease, died on Sunday, ...

Background: Bcl2-associated athanogene 3 (BAG3) is a multifunctional adaptor protein that regulates diverse cellular functions. Mutations in BAG3 are causally linked to myofibrillar myopathy and ...

Success in obtaining orphan drug (a drug used for treating rare disease) designation will accelerate the approval process of the new drug, including speeding up of the review process, waiver of the ...

HONG KONG, Jan. 10, 2024 /PRNewswire/ -- The Centre for Chinese Herbal Medicine Drug Development of Hong Kong Baptist University (HKBU) has developed a new drug using effective components of a Chinese ...

of Hong Kong Baptist University (HKBU) has developed a new drug using effective components of a Chinese herbal medicine, Chaenomelis Fructus, for the treatment of the rare disease, myofibrillar ...