Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.

A 36-week treatment regimen of pegcetacoplan reduces geographic atrophy growth rate while demonstrating a favorable safety profile.

Now, researchers from University of California San Diego and their colleagues report that they have identified a key pathway ...

Akari Therapeutics, Plc (Nasdaq: AKTX), an innovative biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, announces shareholder approval in connection with ...

OCU410 is being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 2-3 million people in the U.S. & EU. Current ...

By Dr. Kaushik Mandal Rare diseases very often remain in the shadows, as common people have limited awareness about them.

A rare spinal surgery in Thiruvananthapuram, India, has transformed the life of 23-year-old Mohamad Raishan Ahmed from the ...

The spinal defect of a 23-year-old Maldivian was cured through a rare surgery at a private hospital in Thiruvananthapuram ...

The company is developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including inherited retinal diseases ("IRDs"), such as RP, LCA, ...

Bethel University is hosting a charity basketball game Saturday to raise funds for a local teenage girl with spinal muscular atrophy, or S.M.A.

MRI has shown that increases in the white matter of the brain and ventricle volume may be risk factors for the progression of ...

Israeli researchers discover that better glycemic control can improve overall health of the brain and slow age-related ...