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Every story we pull is tagged to the disease it’s about — so you can filter to your condition and read only what matters.
20 earlier
Why process characterization is the key to consistent, safe, and scalable cell therapies.
Original title: “All about cell therapy process characterization”
Advances in genomics, biomarkers ... targeted therapeutic approaches. However, clinical research in rare and ultra-rare diseases presents distinct challenges. Small and geographically dispersed patient populations, limited natural history data and high unmet medical need require carefully designed studies and specialist operational expertise. As drug development continues to evolve across rare and complex diseases, initiatives such as Project Optimus are influencing early-phase trial design, while ... Advances in genomics, biomarkers and data-sharing initiatives have transformed understanding of many rare conditions, enabling earlier diagnosis and more targeted therapeutic approaches. However, clinical research in rare and ultra-rare diseases presents distinct challenges. Small and geographically dispersed patient populations, limited natural history data and high unmet medical need require carefully designed studies and specialist operational expertise. As drug development continues to evolve across rare and complex diseases, initiatives such as Project Optimus are influencing early-phase trial design, while advances in cell therapies, including emerging CAR T-cell approaches, continue to show how innovation can translate into meaningful patient outcomes. Rare Disease Day 2026 highlights how clinical research is advancing diagnosis, trial design and treatment development for rare and ultra-rare diseases. For clinical research organisations and healthcare partners, Rare Disease Day is a timely opportunity to reflect on how research, collaboration, and patient-centred trial design can help translate awareness into meaningful progress. Delivering clinical trials in rare and ultra-rare diseases requires flexibility, specialist knowledge and close collaboration with patients, investigators and advocacy groups. Trial designs must balance scientific rigour with the realities faced by people living with rare conditions, with a clear focus on accessibility and minimising patient burden.
Original title: “How Clinical Research Advances Rare Disease Treatment on Rare Disease Day 2026 | Simbec-Orion”
optimal strategies for the surgical and long term management of malignant struma ovarii
Original title: “Optimal Strategies for the Surgical and Long-Term Management of Malignant Struma Ovarii.”
orbital mri for diagnosing giant cell arteritis in cases of anterior ischaemic optic neuropathy
Original title: “Orbital MRI for diagnosing giant cell arteritis in cases of anterior ischaemic optic neuropathy.”
association analysis of hsf1 variable number tandem repeat expansion and coding variants with essential tremor risk in a large cohort
Original title: “Association Analysis of HSF1 Variable Number Tandem Repeat Expansion and Coding Variants with Essential Tremor Risk in a Large Cohort.”
seasonality of igg4 related disease
Original title: “Seasonality of IgG4-related disease.”
therapeutic hopes for cftr related recurrent pancreatitis a case series
Original title: “Therapeutic hopes for CFTR-related recurrent pancreatitis: a case-series.”
peripheral ulcerative keratitis puk in autoimmune diseases a severe ocular manifestation not always associated with systemic disease activity
Original title: “Peripheral ulcerative keratitis (PUK) in autoimmune diseases: a severe ocular manifestation not always associated with systemic disease activity.”
need for improvement in healthcare provision for rare endocrinological diseases in germany using the example endogenous cushing s syndrome
Original title: “[Need for Improvement in Healthcare Provision for Rare Endocrinological Diseases in Germany Using the Example Endogenous Cushing's Syndrome].”
management of acute acquired comitant esotropia using prisms and vision therapy
Original title: “Management of acute acquired comitant esotropia using prisms and vision therapy.”
endoscopic transclival decompression for ventral brainstem tuberculous abscess illustrative case
Original title: “Endoscopic transclival decompression for ventral brainstem tuberculous abscess: illustrative case.”
several considerations on the pathology treatment and prognosis of laryngeal nut carcinoma
Original title: “Several Considerations on the Pathology, Treatment, and Prognosis of Laryngeal NUT Carcinoma.”
current landscape of clinical trials for mrna based therapeutics
Original title: “Current landscape of clinical trials for mRNA-based therapeutics.”
ultrasound characteristics of budd chiari syndrome a multi centre analysis
Original title: “Ultrasound Characteristics of Budd-Chiari Syndrome - A Multi-Centre Analysis.”
atypical presentation of pediatric spinal tuberculosis as chronic abdominal pain a case report
Original title: “Atypical Presentation of Pediatric Spinal Tuberculosis as Chronic Abdominal Pain: A Case Report.”
intraventricular neurocytoma presenting as kleine levin syndrome resolution after resection and review of neurosurgical conditions associated with hypersexuality illustrative case
Original title: “Intraventricular neurocytoma presenting as Kleine-Levin syndrome: resolution after resection and review of neurosurgical conditions associated with hypersexuality. Illustrative case.”
telomeric repeat containing rna g quadruplexes trigger zbp1 mediated cell death
Original title: “Telomeric repeat-containing RNA G-quadruplexes trigger ZBP1-mediated cell death.”
understanding treatment care and delays in acquired hemophilia a a population based study from nova scotia canada
Original title: “Understanding Treatment Care and Delays in Acquired Hemophilia A: A Population-Based Study From Nova Scotia, Canada.”
subacute sclerosing panencephalitis a case report of atypical long term survivor
Original title: “"Subacute sclerosing panencephalitis: a case report of atypical long-term survivor".”
exploratory whole exome sequencing identifies candidate dna variants in ocular behcet disease a pilot study from a pakistani cohort
Original title: “Exploratory whole-exome sequencing identifies candidate DNA variants in Ocular Behcet disease: a pilot study from a Pakistani cohort.”