SAN DIEGO, Calif. – Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the journal Med published results from the open-label study, DAFFODIL™, evaluating the safety, tolerability and exploratory efficacy of DAYBUE® (trofinetide) in girls ages two to four living with Rett syndrome (n = 15). The long-term Phase 2/3 study demonstrated DAYBUE...
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London, UK – A drug used to treat rheumatoid arthritis could also prevent the disease in individuals deemed to be at risk. Results from a Phase 2b clinical trial, published today in The Lancet by researchers led by King’s College London, provides hope for arthritis sufferers after it was shown...
Clinical trial diversity is not a new concept–the U.S. Food and Drug Administration (FDA) issued a draft guidance providing specific recommendations to industry on how to improve diversity in clinical trials in April 2022 but the passage of the Food and Drug Omnibus Reform Act, or FDORA, highlighted that the...
Santa Barbara, Calif. — It’s official: The ketogenic diet proved to be effective at controlling polycystic kidney disease (PKD) in the first randomized controlled clinical trial of ketogenic metabolic therapy for PKD. “I’m really happy about these clinical trial results,” said UC Santa Barbara biologist Thomas Weimbs, whose lab was...
London, England – A collaboration of researchers, led by UCL and Great Ormond Street Hospital, have published successful results from a Phase II clinical trial for the treatment of BRAF mutated low-grade paediatric gliomas. Gliomas are cancerous brain tumours that start in glial cells – the supporting cells of the...
PLEASANTON, Calif. & NANJING, China – IASO Biotherapeutics (IASO Bio) and Innovent Biologics, Inc. (Innovent, HKEX: 01801) today announced that the results of initial clinical studies on treatment of relapsed or refractory multiple myeloma (RRMM) in subjects using fully human BCMA-targeting CAR (CT103A), an investigational chimeric antigen receptor (CAR) T-cell...
Boston, Mass. – An international, phase 3 clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare disease that can cause debilitating symptoms. The study, published in the New England Journal of Medicine, found that treatment with inebilizumab greatly reduced the symptoms of immunoglobulin...
People living with rare diseases often find themselves with more questions than answers when it comes to diagnosis and treatment. Symptoms can be unclear, making misdiagnoses common. This is often the case for people living with WHIM syndrome – a rare and difficult-to-diagnose disorder in which the body’s immune system...
Burton-on-Trent, England – Clinigen, the global pharmaceutical services company, today launched a new campaign called ‘What is Possible?’ to help empower patients and medical professionals to ask what more is possible in the treatment of rare diseases. There are over 7,000 rare diseases impacting over 300 million people globally, and...
CLINUVEL PHARMACEUTICALS LTD today announced that it has received the necessary regulatory and ethics committee approvals to commence a new study in patients with the rare DNA repair disorder Xeroderma Pigmentosum (XP). The Phase II study (CUV156) will evaluate the safety of SCENESSE® (afamelanotide 16mg)¹ in XP-C patients, as well...