Angelman Syndrome News

July 28, 2021July 28, 2021

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other ... Read more

July 21, 2021July 21, 2021

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

Angelman Syndrome News

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies ... Read more

July 14, 2021July 14, 2021

Nominations Open for 2022 Eurordis Black Pearl Awards

Angelman Syndrome News

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work ... Read more

July 7, 2021July 7, 2021

FAST Supports UPenn Research Into microRNA as Angelman Therapy

Angelman Syndrome News

The Foundation for Angelman Syndrome Therapeutics (FAST) has opened a program supporting new research into treating Angelman syndrome by using microRNA (miRNA) to boost UBE3A protein levels. FAST will collaborate ... Read more

July 1, 2021July 1, 2021

Centogene Sets Mission to Cure 100 Rare Diseases in Next 10 Years

Angelman Syndrome News

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, ... Read more

June 30, 2021June 30, 2021

Trial for GTX-102 to Open Enrollment Soon in the UK

Angelman Syndrome News

The United Kingdom’s Medicines Healthcare Products Regulatory Agency (MHRA) has authorized a Phase 1/2 clinical trial to test the investigational therapy GTX-102 in children and adolescents with Angelman syndrome. The open-label KIK-AS trial (NCT04259281), which ... Read more

June 23, 2021June 23, 2021

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

Angelman Syndrome News

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health ... Read more

June 23, 2021June 23, 2021

Neuren Closer to Phase 2 Testing of NNZ-2591 After FDA Guidance

Angelman Syndrome News

Neuren Pharmaceuticals is a step closer to Phase 2 clinical testing of NNZ-2591, its investigational therapy for Angelman syndrome, Phelan–McDermid syndrome, and Pitt Hopkins syndrome, after the company received clear ... Read more

June 16, 2021June 16, 2021

Get Ready for NORD’s Patient and Family Forum, Set for June 26-27

Angelman Syndrome News, COVID-19 News

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the ... Read more

June 16, 2021June 16, 2021

Jacob Family Pledges Up to $250K to FAST for Donation Matches

Angelman Syndrome News

Philanthropists Ryan and Annie Jacob have pledged up to $250,000 to the Foundation for Angelman Syndrome Therapeutics, known as FAST, through a new donation matching program. The Austin, Texas-based couple, ... Read more

June 9, 2021June 9, 2021

Rare 2030 Action Campaign Pushing for European Plan for Rare Diseases

Angelman Syndrome News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in ... Read more

June 2, 2021June 2, 2021

Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill

Angelman Syndrome News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in ... Read more

June 2, 2021June 2, 2021

KIK-AS Phase 1/2 Trial of GTX-102 Can Resume Enrollment in Canada

Angelman Syndrome News

Health Canada has given the green light for a Phase 1/2 clinical trial to resume enrollment to test the investigational therapy GTX-102 in children and adolescents with Angelman syndrome in Canada. This ... Read more

May 26, 2021May 26, 2021

Foundation Funds Development of Cell Models to Advance Research

Angelman Syndrome News

A newly funded research project seeks to create cellular models that can be used to study two of the less common types of genetic abnormalities that cause Angelman syndrome. The ... Read more

May 19, 2021May 19, 2021

Pro Racer Cameron Lawrence Will Fuel FAST Awareness at NASCAR

Angelman Syndrome News

The Foundation for Angelman Syndrome Therapeutics, known as FAST, will get a high-octane boost when professional racecar driver Cameron Lawrence makes his debut at the upcoming NASCAR Camping World Truck ... Read more