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

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

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 ...

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 ...

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. ...

These gorgeous felines go by many names. Some call it ‌a Red Point Siamese, Fire Tip Siamese, or Red Lynx Point Siamese.

The best vaginal moisturizers have a pH similar to the vagina’s natural range. Discover expert-recommended creams, gels, suppositories, and oils for the vagina and vulva.

A study reveals attrition rates in patients with geographic atrophy on complement inhibitor therapies, highlighting the need for improved treatment adherence strategies. Q&A: Rachel Downes, MD, ...

24.03.2015: Educational case report - O’Sullivan-McLeod syndrome is a rare form of distal spinal muscular atrophy with relatively good prognosis.

Definition: Macroeconomics is the branch of economics that studies the behavior and performance of an economy as a whole. It focuses on the aggregate changes in the economy such as unemployment, ...