Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Showing 61–80 of 2532 stories
Sanofi's acquired drug has shown success in a rare disease trial, highlighting the company's strategic investment in biotech. Corporate investors like Novo Holdings and Eli Lilly are also actively funding biotech startups this year.
Aktis, a radiopharmaceutical specialist, is pursuing an IPO and has secured partnerships and investments from major pharmaceutical companies including Eli Lilly, Merck & Co., Novartis, and Bristol Myers Squibb. This move highlights the growing interest in radiopharmaceuticals within the biotech sector.
BioCryst is acquiring Astria Therapeutics in a cash-and-stock deal to enhance its portfolio with a long-acting preventive therapy for hereditary angioedema. This acquisition reflects a strategic shift in venture firms' preferences as the biotech sector recovers.
In 2024, the biotech sector has seen 47 M&A and IPO deals totaling $53 billion, with 15 exceeding $1 billion. Notably, cancer, rare diseases, and cell therapies are key areas attracting investment.
CAMP4 plans to utilize funds from its upcoming IPO to advance clinical trials and expand its RAP platform for central nervous system diseases. This move signals a commitment to innovation in the biotech sector.
Zevra's CEO Neil McFarlane highlights the company's diverse funding strategies, including royalty financing, equity, debt, and M&A, to support rare disease R&D. This creative approach addresses the lack of interest from big pharma in the rare disease sector.
The House of Representatives has passed HR 658, which aims to amend qualifications for marriage and family therapists in the Veterans Health Administration. This bill is now under consideration in the Senate's Committee on Veterans' Affairs.
The Senate has introduced bill S 3689 to designate the VA clinic in Lafayette, Louisiana, as the 'Rodney C. Hamilton Sr. VA Clinic.' The bill has been read twice and referred to the Committee on Veterans' Affairs.
The biopharma industry is increasingly focused on the weight loss sector, projected to surpass $130 billion by 2035. Analysts anticipate significant developments in the oral market and novel mechanisms as 2026 approaches.
AgomAb Therapeutics successfully priced its IPO at $16 per share, raising $200 million to advance its pipeline of therapies for chronic fibrotic disorders. The offering included 12.5 million American Depositary Shares (ADSs), positioning the company for further development in immunology and inflammatory diseases.
The CDC highlights regions with a high risk of dengue fever, emphasizing the importance of awareness for travelers. This information aims to enhance public health safety and inform preventive measures.
H5 bird flu outbreaks are reported in wild birds, poultry, and sporadic human cases, prompting ongoing monitoring and response efforts. The CDC emphasizes the need for vigilance in both animal and human health sectors.
CDC highlights its initiatives in the Caribbean, focusing on public health efforts in Jamaica and Trinidad & Tobago. The report emphasizes ongoing collaborations but lacks specific policy changes or funding details.
The CDC has released a biography for the acting Director of the Division of High-Consequence Pathogens and Pathology. This update provides insights into the leadership within the agency but lacks significant policy implications.
A recent virtual tumor board discussed the case of a 57-year-old man with hydroxyurea-resistant polycythemia vera, highlighting the need for individualized treatment approaches. The discussion emphasized the significance of clinical trial data in managing myeloproliferative neoplasms.
The article highlights the growing access gap for rare disease therapies, which now represent over 40% of new drug approvals. Despite regulatory adaptations, reimbursement and health technology assessment frameworks lag behind, creating a disconnect between drug approval and real-world availability.
Denali Therapeutics presents promising data on its enzyme transport vehicle technology across three clinical programs for lysosomal storage disorders, including Hunter syndrome. The FDA has selected DNL126 for the START Pilot Program to expedite its development, while tividenofusp alfa (DNL310) shows potential to address the full spectrum of Hunter syndrome ahead of its PDUFA date on April 5, 2026.
FDA has determined the regulatory review period for ALFAPUMP, which is relevant for a patent extension application. This notice is part of the legal requirements for patent claims related to the medical device.
FDA has determined the regulatory review period for the AURORA EV-ICD device, which is necessary for patent extension applications. This notice is part of the legal requirements for patent claims related to medical devices.
FDA has determined the regulatory review period for COBENFY, which is necessary for the patent extension application process. This notice is part of the legal requirements for patent claims related to human drug products.
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