Obtained ex-US Regulatory Clearance for DT-818, a Potentially Best-in-Disease Treatment for Myotonic Dystrophy Type-1 (DM1) Trials of DT-216P2 in Friedreich Ataxia (FA) and DT-168 in Fuchs Endothelial ...

Az Oklahoma City Thunder továbbra is megállíthatatlan a NBA-ben: Shai Gilgeous-Alexander vezetésével 137–106-ra kiütötte a New Orleans Pelicanst, ezzel 7–0-ra fokozta mérlegét, akárcsak tavaly ...

A labdarúgó-NB I 12. fordulójának első vasárnapi mérkőzésén az elmúlt hetekben szenvedő Újpest 3-1-re győzni tudott a Diósgyőr otthonában úgy, hogy egy félidőt emberhátrányban futballozott. Damir ...

Az Európa-bajnok Gian van Veen a Players Championship Finals első fordulójában is összecsaphat a dortmundi döntőben legyőzött világelső Luke Humphriesszel (Fotó: Getty Images) Már csak két nagy ...

In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity Biosciences, to develop several promising experimental therapies for various types of muscular ...

PepGen shifts focus to PGN-EDODM1 for Myotonic Dystrophy Type 1 after discontinuing PGN-EDO51 for Duchenne Muscular Dystrophy. PGN-EDODM1 achieved the highest mean splicing correction to date (53.7%) ...

Background Myotonic dystrophy type 1 (DM1) is a multisystem disorder with autosomal dominant inheritance, caused by the abnormal expansion of the CTG triplet in the DMPK gene. Biomarker discovery in ...

This manuscript presents significant and important work that advances single-molecule imaging technology of transcription with simultaneous analysis of several parameters. However, currently, the ...

Avidity Biosciences (NASDAQ:RNA) shares surged as much as 45% in premarket trading on Monday after Novartis (NYSE:NVS) agreed to buy the biotechnology company in a deal valued at $12 billion.

The takeover of its competitor, announced Sunday, could also bring some attention to Dyne Therapeutics, which has a similar RNA-based pipeline in rare muscle diseases. Dyne Therapeutics stands to reap ...