New York, NY – The novel hemophilia therapeutic marstacimab has been accepted for submission by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for regulatory approval, according to a news release. Pfizer announced in a news release that the Biologics License Application (BLA) for its...
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Leiden, The Netherlands – Pharming Group N.V. announced today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS). Pharming has engaged with the US Food and Drug Administration (FDA) and has received feedback on its plans...
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,...
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...
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...
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...
Cambridge, MA – Studies at Picower Institute and elsewhere are producing mounting evidence that light flickering and sound clicking at the gamma brain rhythm frequency of 40 Hz can reduce Alzheimer’s disease (AD) progression and treat symptoms in human volunteers as well as lab mice. In a new study in Nature using a...
Cambridge, Mass. – Early-stage trials in Alzheimer’s disease patients and studies in mouse models of the disease have suggested positive impacts on pathology and symptoms from exposure to light and sound presented at the “gamma” band frequency of 40 Hz. A new study zeroes in on how 40Hz sensory stimulation helps...
By Raffaele Pereno – PhD, MBA VIENNA, Austria – A discussion with AATech CEO Rüdiger Jankowsky at BioEurope highlighted a decisive shift in how alpha‑1 antitrypsin (AAT) may be deployed in respiratory medicine. While most companies in the field continue to focus on treating the rare genetic disorder alpha‑1 antitrypsin...
Philadelphia, January 2, 2024 – Findings from a pioneering study in The American Journal of Pathology, published by Elsevier, reveal that administration of the neuropeptide α-melanocyte–stimulating hormone (α-MSH) promotes corneal healing and restores normal eye function to an otherwise degenerating and diseased cornea by providing protection against cell death and...