Attralus has raised $116 million to support the clinical development of AT-01, its diagnostic imaging agent for systemic amyloidosis, a group of rare conditions that includes familial amyloid polyneuropathy (FAP). Proceeds from the Series B financing will also be used to develop therapeutic agents for different forms of systemic…
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…
The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation RNA interference (RNAi) therapy candidate for familial amyloid polyneuropathy (FAP). Hereditary ATTR (hATTR) amyloidosis, which includes FAP, “is a rare, rapidly progressive, debilitating and fatal condition and we are…
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…
Vutrisiran, an investigational therapy for familial amyloid polyneuropathy (FAP), eased neurological impairments, lessened heart stress, and ameliorated quality of life, nutritional status, and social engagement in adult patients, according to new nine-month data from HELIOS-A trial. “These additional data from the HELIOS-A study show the potential of vutrisiran…
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…
Long-term treatment with Tegsedi (inotersen) helps preserve quality of life in people with familial amyloid polyneuropathy (FAP) for up to three years, according to data from the Phase 3 NEURO-TTR trial and its extension study. While patients initially assigned to a placebo stopped experiencing declines in quality of…
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…
Changes in the function of the retina, in combination with nerve damage in the cornea, can be used as early biomarkers of hereditary transthyretin (hATTR) amyloidosis, also known as familial amyloid polyneuropathy (FAP), a study reports. Researchers suggest that a complete eye exam could be useful to detect problems…