"The acceptance of our Fragile X syndrome OLE data as a podium presentation at AAN underlines the significant unmet need in FXS, and the potential for ZYN002 to become the first-and-only approved ...

for the treatment of FXS and for the treatment of 22q11.2 deletion syndrome (22q). Additionally, ZYN002 has received FDA Fast Track designation for the treatment of behavioral symptoms in patients ...

for the treatment of FXS and for the treatment of 22q11.2 deletion syndrome (22q). Additionally, ZYN002 has received FDA Fast Track designation for the treatment of behavioral symptoms in patients ...

I made notes in his medical records, which I revisited a few years later when it was confirmed that DiGeorge syndrome was "located" on the 22nd chromosome (22q11). This discovery led to it being ...

Background The recurrent ∼600 kb 16p11.2 BP4-BP5 deletion is among the most frequent known genetic ... birth asphyxia, fetal alcohol syndrome, and/or prematurity <30 weeks. In the European series, ...

Tree man syndrome, or epidermodysplasia verruciformis (EV), is a rare, incurable skin disorder that makes people highly susceptible to human papillomavirus (HPV) infection. The disease can lead to ...

From reproductive rights to climate change to Big Tech, The Independent is on the ground when the story is developing. Whether it's investigating the financials of Elon Musk's pro-Trump PAC or ...

Wernicke-Korsakoff syndrome is a neurological condition caused by a long-term deficiency of vitamin B1 (thiamine). It involves two brain disorders that often occur together: Wernicke encephalopathy ...

Celine Dion is "getting back into the swing of things" after being diagnosed with stiff person syndrome in 2022. However, the singer – who turns 57 on March 30 – first noticed symptoms in 2008 ...

For anyone trying to win or hold onto power, the only thing more debilitating than not knowing your enemy is persuading yourself that you have no enemy. Or, as is the case for President Donald ...

The Food and Drug Administration approved a medication this week known as VYKAT XR to treat hyperphagia — or an incessant feeling of hunger — in kids and adults with Prader-Willi syndrome. The ...