Fragile X News Today

September 17, 2021September 17, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 10, 2021September 10, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Fragile X News Today

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 3, 2021September 3, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Fragile X News Today

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

September 3, 2021September 3, 2021

Anavex2-73 Shows Efficacy in Fragile X Mouse Study, Phase 2/3 Trial Planned

Fragile X News Today

Treatment with Anavex2-73 (blarcamesine) safely and significantly eased behavioral symptoms of fragile X syndrome (FXS), and normalized some aspects of brain chemistry, in a preclinical study in mice. Anavex Life ... Read more

August 27, 2021August 27, 2021

CDC Funds Five-year Study to Better Define Fragile X Natural History

Fragile X News Today

A Rush University Medical Center scientist will use a $4 million “cooperative agreement” from the Centers for Disease Control and Prevention (CDC) to better define the natural history of fragile ... Read more

August 27, 2021August 27, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Fragile X News Today

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 20, 2021August 20, 2021

EU Orphan Drug Status Sought for Nova Therapy Using Hallucinogenic Psilocybin

Fragile X News Today

Nova Mentis is seeking orphan drug designation from the European Medicines Agency (EMA) for psilocybin, its investigational therapy for fragile X syndrome. Psilocybin, found naturally in certain mushrooms — popularly ... Read more

August 18, 2021August 18, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Fragile X News Today

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 13, 2021August 13, 2021

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

COVID-19 News, Fragile X News Today

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for ... Read more

August 6, 2021August 6, 2021

Advocates Lobby US Congress During Virtual Rare Disease Week

COVID-19 News, Fragile X News Today

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the ... Read more

August 6, 2021August 6, 2021

Synaptogenix, Nemours Strike Deal on Bryostatin-1 Trial

Fragile X News Today

Synaptogenix and Nemours A.I. DuPont Hospital for Children have signed an agreement to collaborate on a clinical trial to test the investigational medication bryostatin-1 in people with fragile X syndrome ... Read more

July 30, 2021July 30, 2021

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

Fragile X News Today

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 30, 2021July 30, 2021

FRAXA Funds Study of High-fat, Low-carb Diets on Fragile X Seizures

Fragile X News Today

A $90,000 grant from the FRAXA Research Foundation will fund a preclinical study that aims to assess the potential benefits of a diet that is high in fat and low ... Read more

July 23, 2021July 23, 2021

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

Fragile X News Today

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 23, 2021July 23, 2021

Social Stigma Against Fragile X Used For Political Gain in Rural Cameroon

Fragile X News Today

In a rural village in Cameroon, in West Africa, social stigma against those with intellectual disabilities such as fragile X syndrome exists in an uneasy relationship with a deeply ingrained ... Read more