A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…

Differences in pre-pandemic mother-daughter relationships, and social and adaptive behavior significantly predicted worries and impact of the COVID-19 pandemic in school-aged girls with fragile X syndrome (FXS), a U.S. study reports. Notably, this association pattern was distinct from that of matched girls without the condition, whose responses were determined…

The ABLE Employment Flexibility Act and better workplace opportunities for people with disabilities are among priorities for this year’s National Fragile X Foundation (NFXF) Advocacy Day, set for March 1. For more than 20 years, the nonprofit organization has brought advocates together in Washington, D.C., to meet…

Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…

Many COVID-19 clinics are large, crowded, and on the boisterous side, which can be overwhelming for people with fragile X syndrome and other neurodevelopmental disorders needing to go there for vaccination. Such concerns led the UC Davis MIND Institute in Sacramento, California, to open a custom-designed clinic for vaccine administration. The…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

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 National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

COVID-19 lockdowns posed additional challenges for mothers and their children — from toddlers to teens — with fragile X syndrome, who saw their sleep habits and well-being disrupted, a small survey in Italy revealed. The findings highlight the need to give specific support to mothers and their children with special…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…