We met the primary endpoint for our Ph2b study in PBC with setanaxib! A great milestone for Calliditas, but even greater to see the growing potential of our first-in-class NOX inhibitor in treating rare diseases
Philippa Wyndham Jones’ Post
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Cutaneous adverse effects of imatinib are common and well documented in the literature. However, imatinib-induced lichen planus is an uncommon side effect with only four reported cases. In addition, reactivation of hepatitis B is an extremely rare association. We report two cases of imatinib-induced lichen planus, one of which is associated with hepatitis B reactivation, and one case of lichenoid eruption caused by imatinib. The aim of our report is to make physicians aware of this rare association between lichen planus and imatinib. We also recommend screening for hepatitis B virus before starting treatment with imatinib to avoid serious complications.
Imatinib‐induced lichen planus and lichenoid drug eruption: A report of three cases
onlinelibrary.wiley.com
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Explore a rare case of laryngeal mycosis in an immunocompetent patient. This case report sheds light on the diagnostic and therapeutic challenges in managing fungal infections of the larynx. #LaryngealMycosis #Immunocompetent #cureus 🔗 Read more: https://bit.ly/3uRGApE
Laryngeal Mycosis in an Immunocompetent Patient: A Case Report
cureus.com
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【Glycyrrhizic Acid-Loaded Poly-ɛ-Caprolactone Nanoparticles Decrease PRRSV Infection in MARC-145 Cells】 Full article: https://lnkd.in/gER75FDV (Authored by Samantha Jardon, et al., from UNAM-FESC (Mexico), etc.) Porcine reproductive and respiratory syndrome (PRRS) is an economically devastating disease with worldwide distribution caused by Betaarterivirus suid (#PRRSV). The causative agent Betaarterivirus suid (PRRSV) shows great genetic and antigenic variability with a marked increase in virulence. This study evaluates the in vitro effect of the antiviral content in poly-ε-caprolactone nanoparticles containing #glycyrrhizic_acid (PNPsGA) as a new therapeutic strategy for treating PRRSV infections. #Cytotoxicity #Cell_Morphology #Polymeric_Nanoparticles
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The editors essential reading in CMGH: RASSF4 Attenuates Metabolic Dysfunction-Associated Steatotic Liver Disease Progression via Hippo Signaling and Suppresses Hepatocarcinogenesis These findings suggest RASSF4 as a therapeutic target for MASLD and HCC https://lnkd.in/g3VVUdDa
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Our latest article about the TRAIN-HEART consortium and its contribution to RNA-based therapies to treat ischemic heart diseases is finally out!!
Harnessing the power of RNA therapeutics in treating ischemic heart failure: the TRAIN-HEART story
frontiersin.org
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A new gene-silencing tool shows promise as a future therapy against prion diseases and paves the way for new approaches to treating disease. | Click below to read the full article on Sunalei
CHARMed collaboration creates a potent therapy candidate for fatal prion diseases
https://sunalei.org
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As you may have heard by now, the FDA is continuing to take steps to advance treatments for rare disease. The CBER and CDER together have initiated the START Pilot Program which allows chosen companies with rare disease treatment options to have easier access to FDA support, guidance, and initiatives. Among the rare diseases being studied are: Friedrich's ataxia, Vanishing White Matter disease, Sanfilippo syndrome, NGLY1 Deficiency, Isolated methylmalonic acidemia, Canavan Disease, and Rett Syndrome. We are looking forward to seeing the progress made through the START Pilot Program! #clinicalresearch #innovation #FDA #STARTpilotprogram #gsclinical
FDA Opens Doors for More Treatments for Rare Diseases
fda.gov
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The CAR T-cell therapy targets the CD19 protein ubiquitous on the surface of B cells, which are known to mediate various types of autoimmune diseases, including #MS. #TCellTherapy #AutoimmuneDisease https://lnkd.in/giBpf5Vr
Kyverna’s CAR T-Cell Therapy Candidate Cleared for Phase 2 Trial in Progressive MS
managedhealthcareexecutive.com
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✨✨ Welcome to read the latest research "Convalescent #Plasma Therapy, Therapeutic Formulations of Repurposed Drugs in 20th Century Epidemics against #COVID-19: A Systematic Review" by Dr. Diego Fernández Lázaro et al. 🔗 Paper link: https://lnkd.in/giMxEvnP Convalescent plasma (CP) therapy has been used successfully in other viral outbreaks in the 20th century and it is old strategies for treating infectious diseases. In this study, we systematically evaluated the effect and safety of CP therapy on hospitalized COVID-19 patients. The results determined that in hospitalized COVID-19 patients the administration of CP therapy with a volume between 200–500 mL and a single transfusion performed in 1–2 h, compared to the control group, decreased viral load, symptomatology, the period of infection, and mortality, without serious adverse effects. CP did influence clinical outcomes and may be a possible treatment option, although further studies will be necessary.
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📊 Ipsen just shared late-breaking data on #elafibranor in #PBC. 👉 Read more in their press release
News from #EASL. We just shared late-breaking data on long term efficacy and patient reported outcomes from our ELATIVE study in #PrimaryBiliaryCholangitis (PBC), a rare cholestatic liver disease.
Ipsen presents long-term elafibranor efficacy and itch-related quality of life data in patients with primary biliary cholangitis
https://www.ipsen.com
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