Ultragenyx Pharmaceuticals RARE announced that the FDA has granted the Breakthrough Therapy designation to its ...

Taysha Gene Therapies, Inc. (NASDAQ:TSHA) is one of the 10 best healthcare penny stocks to buy according to analysts.

In a conversation with People magazine in 2024, Colin Farrell opened up for the first time about his son's rare condition: ...

Angelman syndrome is a rare condition that causes problems with growth and development. It’s caused by a genetic mutation (a change in your genes) that affects the nervous system. It’s named ...

More information: Jacqueline Fátima Martins de Almeida et al, Molecular aspects of Angelman Syndrome: Defining the new path forward, Biomolecules and Biomedicine (2025). DOI: 10.17305/bb.2025.11724 ...

The drug is being developed to treat Angelman syndrome by restoring the natural balance of brain function. Neuren has received Orphan Drug Designation for NNZ-2591 from both the US FDA and EMA.

Angelman syndrome is a rare neurogenetic disorder affecting approximately 1 in 15,000 live births or 500,000 people globally. It results from the loss of function of the UBE3A gene located on the ...

Angelman syndrome diagnosis of Broken Hill baby sparks Huxley's Heroes marathon across NSW By Coquohalla Connor, Sarah McConnell and Patrick Reincke ...

The drug is being developed to treat Angelman syndrome by restoring the natural balance of brain function. Neuren has received Orphan Drug Designation for NNZ-2591 from both the US FDA and EMA.

"Angelman syndrome is a complex neurodevelopmental disorder resulting from the loss of function of a specific gene called UBE3A, which plays a crucial role in brain development," Dr. Issac ...