Myotonic dystrophy type 1 incurs high healthcare costs, driven by age and comorbidities like cardiac and respiratory issues. Early detection and integrated management of multisystemic complications ...

Comedian Gilbert Gottfried died on Tuesday after a long illness, his family announced. His publicist revealed in a statement to multiple publications, including People, that Gottfried had “recurrent ...

Gilbert Gottfried’s family is asking for the late celebrity's fans to show their support by donating money for research of the disease that took his life. On Wednesday, a tweet shared from the ...

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AOC 1001 continues to be generally well tolerated; Avidity provides more information on the rare serious adverse event in a single participant that led to the partial clinical hold AOC 1001 top-line ...

Please provide your email address to receive an email when new articles are posted on . The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals ...

Please provide your email address to receive an email when new articles are posted on . The FDA has granted breakthrough therapy designation to a proprietary oligonucleotide conjugate for the ...

- Patient-reported outcomes support clinical meaningfulness of improvements in function and strength - - Meaningful improvements in overall disease burden reported by both patients and physicians - ...