To establish a public health infrastructure that advances health equity and improves the lives of people with epilepsy, the Centers for Disease Control and Prevention (CDC) has awarded $17.5 million to the Epilepsy Foundation and its partners. The five-year award will support efforts by these organizations to establish and sustain…
A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.
A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
Longboard Pharmaceuticals is planning to launch a Phase 1b/2a clinical trial early next year that will evaluate the company’s investigational therapy LP352 in adults with Dravet syndrome and other types of developmental and epileptic encephalopathies (DEEs). The trial will test the therapy’s effectiveness in lessening seizures in patients…
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 therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
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 an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
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 in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
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 Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
A total of $50,000 has been raised though a Zogenix photo contest to support the Dravet Syndrome Foundation (DSF) and the Epilepsy Foundation. The company’s donation, to be equally divided, was made possible through the people who took part in the Fintepla Photo Diary, a Zogenix effort…
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