A recent study published in PubMed provides clinical and molecular characterization of Brazilian patients with chronic granulomatous disease. This research enhances understanding of the disease's genetic and clinical profiles in this population.

A phase 2 trial evaluated resminostat as a maintenance treatment for patients with advanced-stage mycosis fungoides or Sézary syndrome. The study was multicenter, double-blind, and randomized, providing insights into the drug's efficacy in this rare skin lymphoma.

A recent study published in PubMed details the clinical characteristics and outcomes of adult patients with Hafnia alvei bacteremia. The findings contribute to the understanding of this rare infection and its impact on patient health.

A case report details a patient with Rasmussen's encephalitis who experienced a decade of refractory epilepsy and hemispheric atrophy. This study contributes to the understanding of the disease's progression and potential treatment challenges.

A case report details extensive intracerebral hemorrhage in a triplet pregnancy following in vitro fertilization, highlighting complications that can arise in such scenarios. This study contributes to the understanding of rare pregnancy-related neurological events.

A rare case study highlights the concurrent presentation of idiopathic intracranial hypertension and bilateral optic perineuritis in a young boy. This unique clinical entity contributes to the understanding of these conditions and their potential overlap.

The CDC reports on the effectiveness of COVID-19 vaccines in children aged 9 months to 17 years, providing critical data for public health policy. This analysis covers the period from August 29, 2024, to September 2, 2025.

Amgen has discontinued development of its eczema drug following disappointing Phase 3 trial results, raising concerns about the viability of new autoimmune therapies competing with Dupixent. This decision reflects ongoing challenges in the dermatology pipeline.

n-Lorem, a nonprofit founded by Dr. Stanley Crooke, develops antisense oligonucleotide therapies for patients with nano-rare diseases, including KIF1A-associated neurological disorder (KAND). The organization provides these treatments free for life, addressing a critical gap in care for patients with rare genetic mutations.

The 23rd International Conference on Duchenne and Becker Muscular Dystrophy recently took place, focusing on advancements and challenges in the field. This event highlights ongoing efforts to improve awareness and support for affected individuals and families.

Researchers have identified a four-marker panel that could potentially detect early-stage pancreatic cancer, a disease known for its high mortality rate. This NIH-funded study aims to improve early diagnosis, which is crucial for increasing treatment options.

AstraZeneca has entered a significant alliance with China's CSPC to develop up to eight next-generation weight loss medications, potentially worth over $18 billion. This deal marks the largest licensing agreement in the obesity treatment space since 2025.

The FDA faces significant challenges due to politicization, which may impact its long-term effectiveness and decision-making processes. An economist highlights that recent staffing and review changes reflect a strong influence from the Trump administration's political agenda.

EMA recommends marketing authorization for Kygevvi (doxecitine and doxribtimine) as the first treatment for thymidine kinase 2 deficiency, a rare genetic disorder. This decision marks a significant advancement for patients with this condition.

The EMA's CHMP recommended six new medicines for approval during its January 2026 meeting. This marks a significant step in the regulatory process for these potential treatments.

EMA recommends extending the indication of Zynyz (retifanlimab) for treating advanced squamous cell carcinoma of the anal canal in adults. This marks a significant step in immunotherapy options for this patient population.

The CDC's Traveler-Based Genomic Surveillance program has exceeded one million voluntary participants, enhancing biosurveillance efforts. This milestone underscores the importance of genomic data in tracking infectious diseases.

Biopharma IPO activity remains subdued with only six companies going public in January, but a recent SPAC raised $172.5 million, exceeding its initial target of $125 million. This indicates cautious optimism in the sector as more companies prepare to enter the market.

The CDC has launched a series of webinars and podcasts focused on fungal diseases, providing access to clinical resources from leading experts. This initiative aims to enhance awareness and understanding of fungal infections among healthcare professionals.

The CDC has released updated data on confirmed norovirus outbreaks submitted to CaliciNet, detailing state-specific and genotype information. This data is crucial for public health monitoring and response strategies.