Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
Be among the first to receive Kisho Intelligence Briefings
Showing 401–420 of 2553 stories
The CDC highlights the prevalence of fatigue in workplaces, emphasizing its potential to cause injuries. This awareness could prompt policy discussions around workplace safety regulations.
The HHS has issued a request for public input on potential changes to the Federal anti-kickback statute and Beneficiary Inducements CMP, particularly regarding direct-to-consumer sales programs by pharmaceutical manufacturers. This could impact how companies engage with patients and structure their marketing strategies.
The Office of Community Services reallocates $2,293,415 in FY24 funds from the Low Income Home Energy Assistance Program to various states, territories, and tribal organizations. This decision impacts the distribution of resources aimed at assisting low-income households with energy costs.
The HHS has issued an advance notice of proposed rulemaking to gather public comments on enhancing the procurement of American-made PPE and essential medicines for Medicare participating hospitals. This initiative aims to strengthen the supply chain and address the costs associated with domestic sourcing.
The Administration for Children and Families is proposing to collect performance measures for Healthy Marriage and Responsible Fatherhood programs, which have been federally funded since 2006. This initiative aims to enhance data collection on program operations and outcomes.
The U.S. Department of Health and Human Services is hosting the inaugural National Conference on Women's Health from March 11-13, 2026, focusing on research and treatment breakthroughs for women's health issues. This event invites non-Federal and private sector organizations to co-sponsor and participate.
The Administration for Children and Families is seeking a 3-year extension for the Head Start Program Information Report, with updates to the reporting instruments. This proposal is currently under review by the Office of Management and Budget.
The Office of Child Support Enforcement seeks a 3-year extension for the Provision of Child Support Services under the Hague Child Support Convention, with no changes to the federally approved forms. This extension is crucial for maintaining ongoing support services in IV-D cases.
FDA has placed a clinical hold on REGENXBIO's trials for RGX-111 and RGX-121 following a brain tumor development in a five-year-old patient. This decision impacts the urgent treatment needs for boys with MPS II, as delays could lead to further neurodevelopmental decline.
FDA has placed a clinical hold on Regenxbio's gene therapy RGX-111 due to the discovery of a brain tumor in a trial participant. This decision has led to a 30% drop in the company's shares, highlighting significant concerns regarding the safety of the treatment for rare childhood diseases.
FDA has placed a clinical hold on Regenxbio's gene therapies RGX-111 and RGX-121 for severe mucopolysaccharidosis type I (MPS I) following an adverse event in a trial participant. This decision impacts the development of these therapies aimed at delivering the IDUA gene to improve patient outcomes.
Argo Biopharma has initiated a phase 2b trial of its siRNA therapeutic BW-20829, targeting patients with elevated lipoprotein(a) levels. This global study marks a significant step in addressing cardiovascular risks associated with Lp(a).
FDA has approved the first human trial of Life Biosciences' ER-100, a gene therapy aimed at rejuvenating retinal cells in patients with age-related eye diseases. This Phase 1 study will focus on conditions such as open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION).
Akari Therapeutics files a new patent for AKTX-102, an antibody-drug conjugate targeting CEACAM5, enhancing its intellectual property portfolio. This move strengthens its pipeline as the company continues to develop innovative therapies.
Erasmus Medical Center is recruiting participants for a natural history study on CAMK2-related synapthopathies, focusing on the role of the CAMK2 gene. This study aims to gather data on the progression and characteristics of these rare conditions.
The MIHRA initiative focuses on gathering patient-rooted insights to shape research in myositis and related conditions. Sponsored by multiple organizations, including Myositis International and The Myositis Association, this project emphasizes qualitative investigations to better understand patient experiences.
The CDC highlights areas at risk for chikungunya transmission, advising U.S. travelers to consider vaccination. This guidance reflects ongoing public health efforts to manage and prevent outbreaks.
The CDC introduces the DOSE-SYS Dashboard to enhance nonfatal overdose syndromic surveillance using electronic health record data. This initiative aims to improve public health responses to overdose incidents.
The CDC has updated its Infant Mortality Dashboard, providing quarterly estimates of infant, neonatal, and postnatal deaths along with death rates for the five leading causes of death. This tool aims to enhance public health policy and inform stakeholders about critical mortality trends.
The CDC launches the STOP Program to recruit and train international public health consultants, aiming to enhance global health initiatives. This program reflects a strategic move to bolster public health expertise worldwide.
Get the week's most important rare disease developments — pipeline moves, policy changes, and funding signals — delivered to your inbox every Monday.
Free weekly briefing. Unsubscribe anytime.