Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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ENA Respiratory has initiated dosing in a Phase 2 study of INNA‑051, a nasal spray aimed at reducing symptomatic viral respiratory infections. The trial will evaluate the efficacy of once-weekly treatment in this indication.
AgomAb Therapeutics, a Belgian biotech focused on TGFβ inhibitors for fibrotic diseases, is set to raise $200 million through its IPO, offering 12.5 million shares priced between $15 and $17. The company is also developing a preclinical candidate for liver cirrhosis.
SpyGlass Pharma sets terms for a $150 million IPO, offering 9.4 million shares priced between $15 and $17. This Phase 3 biotech focuses on developing implants for chronic eye conditions, aiming for a market value of $549 million at the midpoint.
The CDC reports a new recall related to a Salmonella outbreak linked to dietary supplements containing moringa leaf powder, affecting 65 individuals across 28 states. This highlights ongoing safety concerns in dietary supplements and the need for consumer awareness.
A new observational study sponsored by the National Cancer Institute aims to evaluate treatment responses in patients with advanced central nervous system tumors, including various types of gliomas. The study is currently not yet recruiting participants.
The CDC has launched a Nursing Home Data Dashboard featuring a COVID-19 module that tracks the impact on residents and staff in long-term care facilities. This initiative aims to enhance transparency and inform policy decisions regarding nursing home safety.
The CDC is actively conducting and supporting studies aimed at reducing cancer incidence and eliminating health disparities. This initiative underscores the agency's commitment to addressing inequities in cancer care and prevention.
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 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 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 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).
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