Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Recent research highlights the association between TNF inhibitor therapy and overlapping autoimmunity and demyelination syndromes. This study provides insights into potential adverse effects that may impact treatment decisions for patients receiving TNF inhibitors.
A case study highlights endomyocardial calcification associated with pulmonary and systemic hypertension in a neonate diagnosed with multisystem inflammatory syndrome of newborn (MIS-N). This research contributes to understanding the cardiovascular implications of MIS-N in infants.
The National Institutes of Health has submitted a request for review to the Office of Management and Budget regarding the information collection for Autism Spectrum Disorder research. This is part of compliance with the Paperwork Reduction Act.
The Office of Grants Management is seeking a 3-year extension for the ACF Performance Progress Report form, with minor changes aimed at reducing recipient burden. This proposal aligns with federal regulations and reflects feedback from program offices.
The FDA is amending animal drug regulations to reflect application-related actions for new animal drug applications during July to September 2025. These changes aim to enhance the accuracy and readability of the regulations.
The Office of Refugee Resettlement seeks OMB approval for a 3-year extension of the ORR-6 Performance Report data collection, with minor changes to instructions. This extension is necessary due to expired program funding.
Eikon Therapeutics successfully raised over $381 million in its IPO, marking the largest biotech IPO since 2024. This move signals a potential recovery for Bay Area biotech amidst previous funding challenges.
Several states are introducing legislation to regulate compounded medications, particularly those related to weight loss. This could impact patient access and the compounding practices of pharmacies across the country.
The TrumpRx website is set to launch, providing direct-to-consumer access to discounted pharmaceuticals from major companies like Eli Lilly, Pfizer, and Novo Nordisk. The long-term impact of this pricing strategy on the market remains uncertain.
Agomab Therapeutics and SpyGlass Pharma raised a total of $350 million through initial public offerings, marking a significant week for biotech stock issuances. Agomab is advancing a second drug for idiopathic pulmonary fibrosis, with early-stage data expected in 2026.
Eikon Therapeutics raised $381 million in its IPO to advance its lead cancer immunotherapy program currently in clinical trials. The offering included 21.2 million shares priced at $18 each, reflecting strong investor interest in the biotech sector.
AgomAb is set to raise around $120 million through its IPO to advance its Phase 2b trial of ontunisertib for fibrostenosing Crohn’s disease. Additionally, the Massachusetts biotech plans to use its IPO proceeds to support late-stage studies of GB-0985, an anti-TSLP antibody for severe asthma and COPD.
A webinar hosted by Xtalks will focus on the importance of returning patient data in rare disease clinical trials, emphasizing patient-centric research. Experts from Premier Research will discuss responsible practices for sponsors to enhance patient access to health information.
The S 735 bill, aimed at enhancing research, surveillance, prevention, and treatment for sickle cell disease and other heritable blood disorders, has been introduced and referred to the Committee on Health, Education, Labor, and Pensions. This legislative move could impact funding and focus on these conditions.
The House has introduced HR 1796, aimed at enhancing research, surveillance, prevention, and treatment for sickle cell disease and other heritable blood disorders. The bill has been referred to the House Committee on Energy and Commerce for further consideration.
The 'Patients Before Middlemen Act' (S 882) has been introduced and referred to the Committee on Finance. This legislation aims to address the role of intermediaries in healthcare pricing.
HR 1843 aims to amend the Federal Food, Drug, and Cosmetic Act to enhance transparency in generic drug applications. The bill has been referred to the House Committee on Energy and Commerce for further consideration.
The Prescription Drug Transparency and Affordability Act (HR 2450) has been referred to multiple committees for consideration. This legislation aims to address issues related to drug pricing and transparency in the pharmaceutical market.
The Increasing Transparency in Generic Drug Applications Act (S 1302) has been introduced and referred to the Committee on Health, Education, Labor, and Pensions. This legislation aims to enhance transparency in the generic drug application process.
The Hospital Inpatient Services Modernization Act (HR 4313) has been received in the Senate and is currently under review by the Committee on Finance. This bill aims to modernize inpatient services, though specific details on its impact remain to be seen.
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