相关临床试验
604
89 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
1869
进行中（未招募）
36
6.0%
已完成
297
49.2%
Enrolling By Invitation
13
2.1%
尚未招募
40
6.6%
招募中
104
17.2%
暂停
3
0.5%
终止
38
6.3%
Unknown
19
3.1%
撤回
54
8.9%
暂无批准数据
- AI initiatives at Boston Children's Hospital and Children's Hospital of Eastern Ontario are helping to diagnose rare diseases by analyzing clinical and genomic data. - CHEO's ThinkRare algorithm scans electronic health records and has led to 21 new rare disease diagnoses with a 70% success rate so far. - Boston Children's Hospital's reasoning model reviews unsolved cases and suggests disease candidates, significantly speeding up the diagnostic process. - Researchers caution that clinicians must guard against errors produced by these models, but further validation could transform rare disease care.
- Boston Children's Hospital researchers used OpenAI's o3 model to analyze genomes of 376 undiagnosed patients, yielding new diagnoses for 18 children across four disease areas. - The AI-assisted approach achieved a diagnostic yield of nearly 5%, which researchers called a "total game changer" given that these genomes had already been exhaustively analyzed by human experts. - Diagnoses included 10 neurodevelopmental disorders, 4 neuromuscular disorders, 2 sudden deaths, and 2 early childhood psychosis cases, with findings published in NEJM AI. - Experts caution that LLM results still require rigorous human review and that AI tools are meant to support, not replace, medical professionals in complex diagnostic work.
- Twelve years after the Ice Bucket Challenge raised more than $1 billion for ALS research, artificial intelligence is now powering early detection models and voice-cloning tools to improve patient care and quality of life. - ALS remains incurable and fatal, with over 5,000 Americans diagnosed annually and an estimated 30,000 to 35,000 currently living with the disease, according to the CDC. - New gene-targeted therapies have raised hopes of making ALS a livable or chronic condition, with researchers describing the progress as "right at our fingertips." - The Pete Frates Family Foundation and Boston-based AI provider NWN are partnering to accelerate ALS research and preserve patients' voices through advanced technology.
- GlaxoSmithKline discontinued its popular asthma inhaler Flovent in December 2023 to avoid paying an estimated $367.6 million in additional Medicaid rebates under new federal anti-price gouging provisions. - The discontinuation forced families to switch to more expensive alternatives, with 36% of families reporting difficulty paying for replacements and 30% struggling with insurance coverage according to a Boston Children's Hospital survey. - Following a two-year Senate investigation led by Sen. Maggie Hassan, GSK agreed to allow competitor Glenmark Specialty SA to sell a lower-cost generic version starting this month. - The FDA has approved a generic version of Flovent that should restore affordable access to the critical pediatric asthma medication for the 4.6 million children diagnosed with asthma nationwide.
- Scientists have identified the genetic mechanism behind vaccine-induced immune thrombotic thrombocytopenia (VITT), a rare but potentially fatal blood clotting condition linked to adenovirus-based COVID vaccines. - The study published in The New England Journal of Medicine found that VITT occurs in individuals with specific DNA variants whose immune cells produce cross-reactive antibodies against both the adenovirus and PF4, a blood clotting molecule. - All 100 VITT patients studied had one of two specific genetic variants, but required an additional genetic mutation in immune cells to develop the dangerous cross-reactive antibodies. - The findings could enable safer design of future adenovirus-based vaccines by engineering around the specific protein region that triggers the problematic immune response.
- UCLA has been awarded a $7.5 million grant from Aligning Research to Impact Autism (ARIA) to serve as the Clinical Coordinating Center for the IMPACT Network, an international clinical trials initiative for autism and related neurodevelopmental conditions. - The network aims to address critical gaps in autism research by establishing standardized clinical trial infrastructure and focusing on underrepresented populations, including non-verbal individuals and those requiring significant daily assistance. - Each participating site will enroll a minimum of 125 participants for longitudinal study, with standardized phenotyping and biomarker discovery beginning early next year. - The initiative places patients and families at the center of activities, working closely with advocacy organizations to support outreach and enrollment while providing ongoing clinical care through the network.
- Autobahn Labs, a venture studio focused on transforming academic science into drug discovery programs, has announced a strategic partnership with global pharmaceutical company Astellas Pharma Inc. - The collaboration provides Astellas exclusive investment opportunities in selected drug discovery programs from Autobahn's translational research pipeline, which includes early-stage discoveries from leading academic institutions. - Selected research programs will be advanced through newly formed Program Companies with shared equity interests among Autobahn, Astellas, academic institutions, and founding investigators. - Astellas will receive a right of first negotiation for exclusive licenses to further develop and commercialize intellectual property arising from jointly funded programs.
- ARMR Sciences will begin human trials of its fentanyl vaccine in early 2025 with 40 healthy adults at the Centre for Human Drug Research in the Netherlands. - The vaccine works by attaching a carrier protein to a fentanyl-like molecule to generate antibodies that prevent fentanyl from crossing the blood-brain barrier. - Preclinical studies in rats demonstrated the vaccine blocked 92 to 98 percent of fentanyl from entering the brain, with protection lasting 20 weeks. - The vaccine could offer up to a year of protection in humans and represents a paradigm shift from reactive overdose treatments to preventive intervention.
- Researchers at University of Rochester and Harvard Medical School developed CBD-IN, a nano-micelle formulation that successfully crosses the blood-brain barrier and provides neuropathic pain relief within 30 minutes in mice. - The novel delivery system targets only overactive pain circuits while leaving healthy neurons unaffected, avoiding the motor and cognitive side effects commonly seen with conventional pain medications. - CBD-IN works through a novel mechanism independent of traditional cannabinoid receptors, influencing broader electrical and calcium signaling in nerve cells without causing dependency risks. - The formulation maintained effectiveness through repeated use and delivered significantly higher brain concentrations compared to standard oil-based CBD products.
- Ovid Therapeutics announced positive Phase 1 results for OV329, a next-generation GABA-aminotransferase inhibitor, showing 53% inhibition of GABA-AT at 5 mg dose with statistical significance (p=0.0001). - The drug demonstrated superior or comparable efficacy to vigabatrin, the current first-generation GABA-AT inhibitor, while showing a favorable safety profile without ophthalmic complications. - OV329 successfully penetrated the brain and engaged its target, with biomarker studies confirming increased GABA levels and inhibitory activity across multiple validated measures. - The company plans to advance OV329 into Phase 2a trials for drug-resistant focal onset seizures in Q2 2026, targeting a significant unmet medical need in epilepsy treatment.
