US biotech Regenxbio (Nasdaq: RGNX) has successfully completed the BLA submission for its mucopolysaccharidosis II (MPS II) ...

Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...

Denali Therapeutics Inc.’s DNLI share price has dipped by 10.43%, which has investors questioning if this is right time to buy.

Rare diseases affect multiple organ systems and approximately 50–75% of them affect children. Nearly 80% of all rare diseases are estimated to have an underlying genetic aetiology. The Indian Council ...

Some forms, like MPS I (Hurler syndrome) and MPS II, also involve severe neurological decline, while others, such as MPS IV (Morquio syndrome), primarily affect the skeletal system without ...

Instead of a 1-1 count for Muncy, the batter was in an 0-2 hole and struck out three pitches later. Poteet played in Triple-A for part of last season, which is where the ABS system was tested ...

The Milwaukee Brewers' spring training journey has just begun, but one hurler is already being shut ... 277 batting average against and a 1.45 WHIP in 30 1/3 innings across 33 games in three ...

GLENDALE, Ariz. (AP) — Chicago Cubs pitcher Cody Poteet believed the 95 mph fastball he threw to Los Angeles Dodgers slugger Max Muncy was in the strike zone. Plate umpire Tony Randazzo ...