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...
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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...
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...
CHENGDU, China — Clover Biopharmaceuticals, Ltd., a global commercial-stage biotechnology company, today announced positive preliminary safety, efficacy and pharmacokinetics data in a Phase Ⅰ clinical trial evaluating SCB-219M, an innovative thrombopoietin receptor agonist (TPO-RA) mimetic bispecific Fc-fusion protein produced from CHO cells, for the treatment of cancer patients with chemotherapy-induced...
BOULDER, Colo. – Clovis Oncology, Inc. (NASDAQ: CLVS) announced that the first presentation of data from the randomized, Phase 3 ARIEL4 study of Rubraca® (rucaparib) will take place today in an oral presentation at the Society of Gynecologic Oncology Virtual Annual Meeting on Women’s Cancer (SGO). The data demonstrate that Rubraca...
St. Louis, MO – Alzheimer’s disease has plagued one large Colombian family for generations, striking down half of its members in the prime of life. But one member of that family evaded what had seemed would be fate: Despite inheriting the genetic defect that caused her relatives to develop dementia...
Madrid, Spain – A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has made a breakthrough discovery in the understanding of cardiac arrhythmias by unraveling the complexities of Andersen-Tawil syndrome (ATS), an extremely rare inherited cardiac disorder. Led by Dr. José Jalife, head of the CNIC Cardiac Arrhythmia Group,...
Madrid, Spain – Hutchinson-Gilford progeria syndrome (HGPS) is an extremely rare genetic disease that affects just 1 in every 20 million people; it is estimated that fewer than 400 children in the world have the disease. HGPS is characterized by accelerated aging, severe atherosclerosis, and premature death at an average...