Since newborns with phenylketonuria (PKU) do not initially have any symptoms, the first step in treating PKU is timely ...

In a world flooded with cheap facsimiles of human art, will we still be able to rely on our own faculties for creation, or ...

Sarcopenia, which is a progressive and extensive decline in muscle mass and strength, is common with aging and is estimated ...

Spinal muscular atrophy (SMA) is an autosomal recessive genetic disease that causes progressive degeneration of motor neurons, affecting approximately 1 in 10,000 births.

Salanersen (BIIB115/ION306) is a novel antisense oligonucleotide (ASO) with the potential to achieve high efficacy and once yearly dosing in spinal muscular atrophy (SMA)Interim Phase 1 data show ...

Thiruvananthapuram: For the first time in India, pre-symptomatic treatment to an infant, diagnosed with spinal muscular atrophy (SMA) within days of b.

SALT LAKE CITY — In this video, Jay Chhablani, MD, discusses preliminary data from the ArMaDa study of OCU410 gene therapy for geographic atrophy presented at the ARVO meeting.

Boehringer Ingelheim has started a proof-of-concept trial of a drug that could offer the first oral treatment for geographic atrophy (GA), a major cause of irreversible sight loss that affects ...

OTTAWA, Ontario — At the end of World War II, Canada boasted one of the world’s largest navies, with 95,000 uniformed members and 434 ships. The current Royal Canadian Navy is far less ...

Roche’s Evrysdi (risdiplam) tablets have been approved by the European Commission (EC) to treat spinal muscular atrophy (SMA). The new, room-temperature stable formulation of Evrysdi may offer ...

UK start-up Complement Therapeutics has raised an impressive €72 million ($79 million) in first-round financing that will be used to take a gene therapy for sight-robbing disease geographic ...

Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. 2012 Jan;20(1):27-32. doi: 10.1038/ejhg.2011. ...