Broken Hill steps up for the family of a boy born with a rare and debilitating genetic condition. But none will go further ...

Families in Connecticut and beyond are looking for answers when it comes to treating Angelman Syndrome, but fear funding ...

Angelman syndrome shares a lot of symptoms and characteristics with other neurogenetic conditions, like autism, cerebral palsy and Prader-Willis syndrome, according to the Angelman Syndrome ...

Lucas has Angelman syndrome (AS), a rare neurogenetic condition ... could have occurred because both conditions share overlapping symptoms, and cerebral palsy is much more common than AS.

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.

In this study, the transplanted cells would deliver functional Ube3a protein, potentially preventing, halting, or reversing the symptoms of Angelman syndrome. “Our team is very grateful to receive ...

Figure 1. Human heart development stages. The figure shows the development of the human heart by weeks post-conception and corresponding Carnegie stage. In the proximal region of its long arm, human ...

Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide ...