If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation. When ...

BridgeBio said an experimental therapy for a rare form of muscular dystrophy succeeded in a key trial, potentially setting up ...

New interim data from a Phase III trial puts the company on track to file for FDA approval next year in an indication that ...

Blocking a small RNA molecule called miR-140-3b can improve motor function in a mouse model of SMA, a study suggests.

Shares of BridgeBio Pharma rose after the company reported positive data in the phase three study of its treatment of limb-girdle muscular dystrophy. The stock rose 10% to $59.74 in premarket trading ...

The deal, which Novartis expects can “unlock multibillion-dollar opportunities” in the years ahead, hands the company a group ...

ABSTRACT: The present journal paper is the fourth and last part of a review of boxing practitioners’ physiology. The third part has dealt with dietary supplementation, weight control, recovery, and ...

Thomas Crawford, MD, said SAPPHIRE trial data show apitegromab provides clinically meaningful strength gains for children with SMA by targeting muscle in addition to motor neurons. The study enrolled ...

Scholar Rock intends to resubmit the apitegromab Biologics License Application (BLA) upon resolution of Catalent Indiana LLC-related observations Scholar Rock (NASDAQ: SRRK), a global ...

Erin Bates isn't in the clear quite yet after being released from the hospital. The Bringing Up Bates alum revealed that she's lost the function of her right leg after suffering health complications ...

Participants aged 2 to 12 years receiving apitegromab had significantly higher improvements in motor function vs placebo. HealthDay News — For patients with nonambulatory type 2 or type 3 spinal ...