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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…
The first healthy volunteers were dosed in a Phase 1 clinical trial evaluating Inversago Pharma‘s INV-102, a potential therapy for Prader-Willi syndrome (PWS) and other metabolic disorders. According to a company press release, the trial, underway in Canada, started dosing following a “No Objection Letter” from Health Canada.
The U.S. Food and Drug Administration (FDA) is holding a public advisory committee meeting regarding LV-101 (intranasal carbetocin) as a potential treatment of distress behaviors and insatiable hunger in Prader-Willi syndrome (PWS) patients. The Psychopharmacologic Drugs Advisory Committee meeting, to be held virtually Nov. 4 from 10…
One year of treatment with Soleno Therapeutics’ investigational oral therapy diazoxide choline controlled release, known as DCCR, significantly reduced excessive appetite and disease-related behaviors in Prader-Willi syndrome (PWS) patients, according to top-line data from an extension study of the DESTINY PWS Phase 3 trial. These trajectories were significantly…
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…
People with Prader-Willi syndrome (PWS) have smaller pituitary glands than their typically-developing peers, according to data from a small study. The findings also suggest that altered pituitary size is associated with behavioral abnormalities in PWS. Results were published in the journal Biomarkers in Neuropsychiatry, in the study “…
The mean total annual costs of raising a child with Prader-Willi Syndrome (PWS) — an estimate that takes into account services use, impact on parental income, and care arrangements — is more than AU$57,500 (about $42,500) per child, according to an Australian study of four rare disorders that cause…
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…
An $11.2-million, five-year grant has been awarded by the Centers of Excellence in Genomic Science of the National Institutes of Health (NIH) to uncover high-resolution details of the entire human genome. This project should help better understand the genome’s structure and function, as well as provide important clues about the…
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