A Mid-Michigan family is raising money to help their 6-year-old boy with a rare genetic disorder. After spending a few months looking for action figures and fun items, Kristin Emeott spent $500 and ...

Proficio Capital Partners LLC bought a new position in shares of Anavex Life Sciences Corp. (NASDAQ:AVXL – Free Report) in ...

Lucas Oon has Angelman syndrome, which is characterised by a happy demeanour and an easily excitable nature. Read more at ...

From her mother’s perspective, Allison Girouard is an activist in her own right. Although it’s not easy for Amy Girouard-Crush’s daughter to communicate, Allison often “speaks out about ...

Neuren Pharmaceuticals (ASX:NEU) has received a Rare Paediatric Disease Designation from the US FDA for NNZ-2591 in Pitt-Hopkins syndrome and Angelman ...

Learn more about whether Kymera Therapeutics, Inc. or Ultragenyx Pharmaceutical Inc. is a better investment based on AAII's ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

Image caption, Ivor has "come along leaps and bounds" since starting school last year, according to his mum Sarah Sarah, from Monmouth, has a five-year-old son called Ivor who was diagnosed with ...

When Ms. Liu, a cancer patient, picked up a box of medication at the hospital pharmacy, she felt an overwhelming sense ...

The latest Market Talks covering the Health Care sector. Published exclusively on Dow Jones Newswires at 4:20 ET, 12:20 ET ...

Hundreds of people are expected to help shine a light on rare illnesses by taking part in the Rare Disease Fun Run on Sunday, ...