Givinostat is an oral histone deacetylase (HDAC) inhibitor approved by the FDA in 2024 to treat Duchenne patients ages 6 years and older. Pathologic upregulation of HDAC occurs in DMD, leading to ...

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 ...

School of Chemistry, University of Birmingham, Edgbaston, Birmingham B15 2TT, U.K. School of Chemistry, University of Birmingham, Edgbaston, Birmingham B15 2TT, U.K ...

But for 23-year-old Avalon Saph, known to her fans as Lana, her most lucrative gig comes from something far less conventional: selling videos of herself farting. “I’ve been paid like 200 bucks to send ...

The Delhi High Court on Thursday upheld a single judge's refusal of an interim injunction sought by Swiss pharmaceutical giant F Hoffmann-La Roche AG against Natco Pharma Ltd, in a patent dispute over ...