Angelman Syndrome News

May 26, 2020May 26, 2020

AskBio and UNC to Develop Angelman Syndrome Gene Therapy

Asklepios BioPharmaceutical (AskBio) and the University of North Carolina at Chapel Hill (UNC) have entered into an agreement for the development of a gene therapy for Angelman syndrome. Angelman syndrome ... Read more

May 19, 2020May 19, 2020

COVID-19 Delays Clinical Studies of GT-AS for Angelman Syndrome

Angelman Syndrome News, COVID-19 News

PTC Therapeutics is delaying by at least three months the submission of an investigational new drug application requesting that the gene therapy candidate GT-AS enter clinical trials for Angelman syndrome. ... Read more

May 12, 2020May 13, 2020

Ovid CEO Has High Hopes for OV101 to Treat Angelman Worldwide

Angelman Syndrome News

Pharmaceutical industry leader Jeremy Levin believes his company is on the cusp of winning approval for the first medicine that treats the underlying cause of Angelman syndrome. Levin is CEO ... Read more

May 5, 2020May 6, 2020

GTX-102 Placed on Fast Track by FDA for Angelman Syndrome

Angelman Syndrome News

The U.S. Food and Drug Administration (FDA) has given fast track designation to GeneTx Biotherapeutics and Ultragenyx‘s investigational therapy GTX-102 for Angelman syndrome. Fast track is given to speed the development and review of ... Read more

May 4, 2020May 4, 2020

Enrolling Your Child in an Angelman Syndrome Clinical Trial

Angelman Syndrome News

Are you thinking of enrolling your child in a clinical trial for Angelman syndrome? Here’s information that may help you to decide. What is a clinical trial? The goal of clinical ... Read more

April 28, 2020June 1, 2020

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Angelman Syndrome News, COVID-19 News

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their ... Read more

April 27, 2020April 27, 2020

What Are the Different Genetic Variants of Angelman Syndrome?

Angelman Syndrome News

Angelman syndrome is a rare genetic disorder that affects about 1 in 15,000 people. The disease is characterized by intellectual and physical disabilities. There are several variants of Angelman syndrome, ... Read more

April 22, 2020May 23, 2020

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

Angelman Syndrome News, COVID-19 News

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called ... Read more

April 21, 2020April 21, 2020

New Rat Model Of Angelman Syndrome Might Advance Search for Therapies

Angelman Syndrome News

A new rat model of Angelman syndrome (AS) involves complete deletion of the maternal UBE3A gene, which causes multiple behavioral and neurological abnormalities. This model could be useful for better understanding ... Read more

April 17, 2020April 18, 2020

Should Dave Open the Refrigerator? RAREwithCOVID Can Help Decide

Angelman Syndrome News

We have a problem: My husband, Dave, is a surgeon. Normally, we are proud that his long hours go toward an undeniably good cause: to save the lives of children ... Read more

April 17, 2020April 17, 2020

Keeping Your Angel Busy

Angelman Syndrome News

Captain’s Log, Stardate 04.14.2020: This is our 34th day of self-quarantine. To survive, we’ve had to adapt to new restrictions and find distractions to prevent ourselves from losing our marbles. ... Read more

April 16, 2020April 20, 2020

What Special Equipment Will My Child With Angelman Syndrome Need?

Angelman Syndrome News

Angelman syndrome is a complex neurological disorder characterized by symptoms such as scoliosis, walking difficulty, balance issues, behavioral problems, and seizures. If your child is among the 1 in 15,000 ... Read more

April 14, 2020April 14, 2020

Amicus Opens Center to Advance Gene Therapy Programs

Angelman Syndrome News

Amicus Therapeutics has officially opened its Global Research and Gene Therapy Center of Excellence to advance the company’s gene therapy programs for rare diseases such as Angelman syndrome. Located in ... Read more

April 10, 2020April 13, 2020

Understanding the Genetics of Angelman Syndrome

Angelman Syndrome News

Angelman syndrome is a rare neurological disease characterized by physical and mental developmental delays, among other symptoms. The disease affects about 1 in 12,000 to 20,000 people. Most cases of ... Read more

April 7, 2020May 10, 2020

NORD’s Advice on COVID-19: You’re Stronger Than You Think

Angelman Syndrome News, COVID-19 News

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. ... Read more