Researchers identified variants which may cause activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) Results enable clinical genetic testing labs to appropriately reclassify VUSs, accelerating the path to a definitive APDS diagnosis for many patients Findings reveal APDS may be more prevalent than previously estimated Webcast to take place on Monday,...
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Leiden, The Netherlands – Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global, commercial stage biopharmaceutical company developing innovative protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs, in collaboration with Invitae Corporation (NYSE: NVTA, “Invitae”), a leading medical...
ZUG, Switzerland — Pharvaris, a clinical-stage company developing novel, oral bradykinin B2 receptor antagonists to treat and prevent hereditary angioedema (HAE) attacks, today announced the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Investigational New Drug (IND) application for deucrictibant for the prophylactic treatment of...
ZUG, Switzerland, Jan. 27, 2021 – Pharvaris, a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies, including the novel, small molecule bradykinin-B2-receptor antagonists for the treatment of hereditary angioedema (HAE) and other bradykinin-B2-receptor-mediated indications, today announced clinical data from its Phase 1 multiple-ascending-dose study demonstrating...
CLEVELAND, Ohio – University Hospitals (UH) Seidman Cancer Center hematologist-oncologist Leland Metheny, MD, is leading the trial. He says in the two years since the foundational pre-clinical work was completed, the team has shown that it’s feasible to manufacture BAFF CAR T-cells for human subjects. The innovation is introducing genes into...
SYDNEY — Pharmaceutical company Pharmaxis (ASX: PXS; Nasdaq: PXSL) is pleased to announce that additional results of its recently completed international Phase III trial of Bronchitol in patients with cystic fibrosis have been presented at the 2009 European Cystic Fibrosis Conference in Brest, France. The results were presented to the...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that positive results from the Phase 3 OASISplus prospective switch study of donidalorsen in patients with hereditary angioedema (HAE) were published in The Journal of Allergy and Clinical Immunology (JACI) In Practice. Results indicate that patients who switched to donidalorsen from prior...
SAN DIEGO, Calif. – AZD1390, an ataxia telangiectasia mutant (ATM) kinase inhibitor, demonstrated a manageable safety profile in both recurrent and newly diagnosed glioblastoma (GBM) patients when given in combination with standard-of-care radiotherapy and showed preliminary efficacy in recurrent GBM patients, according to results from a global phase I trial presented...
OSLO, Norway — New Investigational Drug Also Shown to Improve Remission Rates and Reduce Short-Term Mortality Compared to Previous Treatments Clavis Pharma ASA (OSE: CLAVIS) today announces positive final results from a Phase II trial of its novel investigational cancer drug, elacytarabine (CP-4055), in patients with late-stage acute myeloid leukaemia...
Lund Sweden – Active Biotech AB (publ) (NASDAQ STOCKHOLM: ACTI) and NeoTX today announced that they have received clearance from the U.S. Food and Drug Administration (FDA) for the Investigational New Drug (IND) application of the tumor targeted superantigen naptumomab estafenatox (Naptumomab). The Phase IIa open label trial will evaluate...