A new clinical decision tool has been developed and validated to predict long-term pain reduction in patients undergoing laparoscopic cholecystectomy for symptomatic cholecystolithiasis. This prospective cohort study aims to enhance patient outcomes by providing tailored predictions.

A recent case study highlights intestinal Behçet disease in a deceased donor kidney transplantation recipient, contributing to the understanding of this rare condition's impact on transplant outcomes. This case adds valuable insights into the complexities of managing rare diseases in transplant settings.

The UK government will integrate the 'Be part of research' service into the NHS App, allowing patients with rare cancers to register for clinical trials. This initiative aims to enhance awareness and inclusivity in clinical research, particularly for brain cancers and other rare diseases.

The EveryLife Foundation for Rare Diseases has announced the 2025 RareVoice Award recipients, recognizing advocates who elevate the rare disease patient voice in policy. This initiative highlights the importance of diverse community representation in advocacy efforts.

Novo Nordisk's early Q4 results exceeded analyst expectations, yet the company faces a projected sales decline for 2026, impacting investor confidence. The 14% slide in stock price reflects concerns over future revenue streams.

Congress has reauthorized the Rare Pediatric Disease Priority Review Voucher (PRV) Program for five years, enhancing support for rare disease research. This legislation is a significant win for the rare disease community, ensuring continued funding and resources.

Savara launches an Early Access Program for molgramostim inhalation solution, aimed at patients with Autoimmune Pulmonary Alveolar Proteinosis. This initiative provides critical access to treatment for a rare disease affecting lung function.

Pfizer's early data on its obesity candidate Metsera has left analysts seeking more information following the company's fourth-quarter earnings call. The limited insights provided have raised questions about the drug's potential and market positioning.

CIRM launches a $100 million initiative called RAPID to accelerate gene therapy research for rare diseases. This program aims to transform the delivery of genetic therapies, which currently are costly and tailored for very few patients.

Pfizer's stock dipped following Phase 2 trial results indicating that its long-acting obesity drug, acquired through a $10 billion deal with Metsera, is 'slightly inferior' to Eli Lilly's leading Zepbound. This development raises concerns about the competitive positioning of Pfizer's new asset in the obesity market.

Rare Disease Day 2026 aims to raise awareness for over 300 million people living with rare diseases, including Prader-Willi Syndrome. The event highlights the advocacy efforts of individuals like Dr. McTighe, who has dedicated her research and training to support those affected by this condition.

Merck highlights its extensive pipeline during its 2025 earnings call, countering claims of modest growth. CEO Robert Davis emphasized the significance of recent deals, positioning the company for future opportunities.

EURORDIS hosted a policy event in recognition of International Childhood Cancer Day, focusing on advocacy for childhood cancer initiatives. The event aimed to raise awareness and promote policy changes to improve outcomes for affected children.

FDA launches the 'PreCheck' program aimed at pharmaceutical facilities that align with national priorities, potentially streamlining the approval process. Meanwhile, Acadia's lupus drug faced setbacks, while MeiraGTx advances its gene therapy portfolio.

Pfizer reports promising mid-stage trial results for PF’3944, demonstrating weight loss of up to 12.3% at 28 weeks. This data positions Pfizer competitively in the obesity treatment market following its acquisition of Metsera.

Daiichi Sankyo has decided to discontinue the development of DS-9606, its next-generation antibody-drug conjugate intended for cancer treatment. This decision reflects a strategic shift in their oncology pipeline, impacting future funding and resource allocation.

Roche has entered a partnership with SangeneBio potentially worth up to $1.7 billion to enhance its RNA interference (RNAi) portfolio. This deal complements Roche's existing RNAi initiatives, including the hypertension candidate zilbesiran, which is partnered with Alnylam.

GSK plans to cut up to 350 jobs in the US and UK as part of its restructuring efforts. This move reflects ongoing adjustments within the company to optimize operations and reduce costs.

Novo Nordisk's next-generation obesity drug, CagriSema, outperformed Wegovy in blood sugar control during a Phase III trial, although it did not achieve the 25% weight loss target. This development could impact the competitive landscape for obesity treatments.

Kahimmune Therapeutics has secured a licensing agreement with Gustave Roussy and SATT Paris-Saclay to develop a vaccine targeting colorectal and pancreatic cancers. This collaboration marks a significant step in advancing innovative cancer therapies.