Medications

Enzyme replacement therapy approved for late-onset Pompe disease

(HealthDay)—Nexviazyme (avalglucosidase alfa-ngpt) was approved for the treatment of the rare inherited disorder late-onset Pompe disease in patients 1 year of age and older, the U.S. Food and Drug Administration announced ...

Diseases, Conditions, Syndromes

A new treatment for rare muscular disease

Rare diseases are sometimes the most difficult to treat because of a lack of research and fewer participants to study.

Medical research

Good news for menopausal women taking hops supplements

Hop-based dietary supplements that many women use to ease the night sweats and hot flashes commonly reported during menopause aren't likely to cause drug interactions, new research from Oregon State University's Linus Pauling ...

Diseases, Conditions, Syndromes

Potential therapy for rare neurologic disease

A targeted therapy, currently being studied for treatment of certain cancers including glioblastoma, may also be beneficial in treating other neurologic diseases, a study at the University of Cincinnati shows.

Medical research

New therapy could treat children with Hunter disease

A team at the University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients ...

Genetics

Biochemists confirm existence of theoretical genetic disorder

Thanks to the sequencing of the human genome, scientists can now discover potential disorders for which there are no known patients. One such disorder is MPS III-E, originally also called Dierks's disorder after its discoverer. ...

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