When your child is diagnosed with a rare genetic disorder such as Sanfilippo syndrome, it can be difficult to…
Articles by Mary Chapman
If your child has Sanfilippo syndrome or mucolipidosis (ML), it is very important that you register them with…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will…
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The…
Lysogene has established a scientific advisory board (SAB) to further explore gene therapy as a treatment for central…
Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania have enhanced their gene therapy…
Siblings of terminally ill children need more support to deal with loss, said the founder of a support website who presented…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about…
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with…
They had a son they adored, and now Cara and Glenn O’Neill longed for a girl. After a couple of…