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 23 Products   150 Diseases   23 Products   167 Trials   15038 News 


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  • ||||||||||  Tysabri (natalizumab) / Biogen, Royalty, Mavenclad (cladribine) / EMD Serono
    Trial primary completion date:  Cladribine Tablets After Treatment With Natalizumab (CLADRINA) (clinicaltrials.gov) -  Dec 2, 2024   
    P4,  N=40, Active, not recruiting, 
    This can allow for interventional radiologists to perform more procedures without exceeding their annual dose limit. Trial primary completion date: Aug 2024 --> Aug 2026
  • ||||||||||  Niktimvo (axatilimab-csfr) / Syndax Pharma, Incyte
    Review, Journal:  Axatilimab: First Approval. (Pubmed Central) -  Nov 28, 2024   
    Axatilimab was added to the NCCN guidelines for cGVHD in August 2024. This article summarizes the development milestones leading to this first approval of axatilimab for the treatment of cGVHD.
  • ||||||||||  Review, Journal:  Neuromuscular diseases: genomics-driven advances. (Pubmed Central) -  Nov 27, 2024   
    Despite these advancements, challenges remain, including the rarity of many NMDs, genetic heterogeneity, and the high costs associated with genomic technologies and therapies. Continued progress in gene therapy, RNA-based therapeutics, and personalized medicine holds promise for further breakthroughs in the management of these debilitating diseases.
  • ||||||||||  Spinraza (nusinersen) / Biogen
    Retrospective data, Journal:  Common complications in spinal muscular atrophy (SMA) type 1 after nusinersen treatment. (Pubmed Central) -  Nov 25, 2024   
    As the survival rate in SMA type 1 increases, the incidence of complications similar to those frequently seen in SMA type 2 and type 3 patients also increases. The follow-up and treatment of patients with SMA should be undertaken by a multidisciplinary team.
  • ||||||||||  Journal:  Benchmarking the medication efficiency and technological progress of diabetes drugs. (Pubmed Central) -  Nov 25, 2024   
    However, the other two GLP-1 drugs, Lixisenatide and Liraglutide, were less efficient...High dose frequency, low HbA1c reduction and insignificant weight loss were the main driving factors behind inefficiencies. Overall, medication efficiency provided an alternative perspective on treatment effectiveness other than conventional measures such as cost-effectiveness.
  • ||||||||||  Zolgensma (onasemnogene abeparvovec-xioi) / Novartis, Evrysdi (risdiplam) / SMA Foundation, PTC Therap, Roche, Spinraza (nusinersen) / Biogen
    Journal:  Use of disease-modifying therapies in spinal muscular atrophy 5q in Mexico (Pubmed Central) -  Nov 23, 2024   
    The Food and Drug administration (FDA) and COFEPRIS (Mexico's Federal Committee for Protection against Sanitary Risks) have approved 3 therapies to increase the production of survival motor neuron (SMN) protein and improve muscle strength and quality of life in patients: nusinersen, onasemnogene abeparvovec xioi, and risdiplam...The most important thing is to establish the diagnosis of the disease and start any of the available treatments to avoid further functional disability and prevent death. The 3 treatments have different mechanisms of action and different adverse effects, and the use of each of them must be individualized according to the patient's profile.
  • ||||||||||  Clinical, Review, Journal:  Lp(a): A Rapidly Evolving Therapeutic Landscape. (Pubmed Central) -  Nov 22, 2024   
    While there are presently no Lp(a)-lowering drugs available for routine clinical use, several promising candidates are currently under investigation. If these prove to be effective in randomized clinical trials, they will expand the cardiovascular care armamentarium and will allow clinicians to treat a presently unmitigated cardiovascular risk factor.
  • ||||||||||  Spinraza (nusinersen) / Biogen
    Enrollment closed:  Spinraza in Adult Spinal Muscular Atrophy (clinicaltrials.gov) -  Nov 20, 2024   
    P=N/A,  N=148, Active, not recruiting, 
    N=25 --> 0 | Recruiting --> Withdrawn Recruiting --> Active, not recruiting
  • ||||||||||  Journal:  Correct use of inhalers for asthma. (Pubmed Central) -  Nov 19, 2024   
    These data will help identify mechanisms to improve patient selection and therapeutic efficacy of Nadofaragene firadenovec. No abstract available
  • ||||||||||  Journal:  Drugs for asthma. (Pubmed Central) -  Nov 19, 2024   
    No abstract available No abstract available
  • ||||||||||  Adstiladrin (nadofaragene firadenovec-vncg) / Ferring, Keytruda (pembrolizumab) / Merck (MSD)
    Review, Journal:  Bacillus Calmette-Gu (Pubmed Central) -  Nov 18, 2024   
    The last years have been exciting regarding new developments in this field after a long period of stagnation. Unfortunately, data available on some alternative therapies are mainly limited mainly to Phase I or II studies with a lack of robust evidence, but a clear trend in future treatments has just been drawn.
  • ||||||||||  Adstiladrin (nadofaragene firadenovec-vncg) / Ferring, Keytruda (pembrolizumab) / Merck (MSD), Anktiva (nogapendekin alfa inbakicept-pmln) / ImmunityBio
    Review, Journal, HEOR, Cost-effectiveness, Cost effectiveness:  Unmet Need in Non-muscle-invasive Bladder Cancer Failing Bacillus Calmette-Gu (Pubmed Central) -  Nov 18, 2024   
    In the rapidly evolving landscape of urothelial cancer, some of these treatment strategies might be cost effective and improve patients' quality of life. The findings of our review highlight the need for novel, more effective therapeutic strategies.
  • ||||||||||  Verquvo (vericiguat) / Bayer, Merck (MSD)
    Review, Journal:  Pharmacological management of heart failure: a patient-centered approach. (Pubmed Central) -  Nov 18, 2024   
    In this context, a patient-centered approach that considers comorbidities and specific clinical scenarios when dosing HF medication is essential. Prevention of hospital admissions for cardiac decompensation is of utmost importance in patients with HF as is the enablement of activities of daily living, an endpoint which has only recently been incorporated into major HF trials.
  • ||||||||||  olpasiran (AMG 890) / Amgen
    Enrollment closed:  A Study to Assess the Effect of Olpasiran on QT/QTc Intervals in Healthy Participants (clinicaltrials.gov) -  Nov 17, 2024   
    P1,  N=32, Active, not recruiting, 
    Prevention of hospital admissions for cardiac decompensation is of utmost importance in patients with HF as is the enablement of activities of daily living, an endpoint which has only recently been incorporated into major HF trials. Recruiting --> Active, not recruiting
  • ||||||||||  Lemtrada (alemtuzumab) / Sanofi
    Retrospective data, Journal:  Infection Risk Associated with High-Efficacy Disease-Modifying Agents in Multiple Sclerosis: A Retrospective Cohort Study. (Pubmed Central) -  Nov 17, 2024   
    Patients initiating heDMAs (natalizumab, alemtuzumab, and ocrelizumab) or meDMAs (interferon beta-1a, interferon beta-1b, fingolimod, teriflunomide, dimethyl fumarate, and glatiramer acetate) were included...In MS, heDMAs were associated with a greater risk of serious infection and UTI compared with meDMAs. These findings suggest the need to carefully monitor and manage the infection risk to optimize the use of heDMAs in MS.
  • ||||||||||  Spinraza (nusinersen) / Biogen
    Review, Journal:  Lumbar Transforaminal Injections of Nusinersen in Spinal Muscular Atrophy Patients. (Pubmed Central) -  Nov 16, 2024   
    Following a review of spinal muscular atrophy pathogenesis and current therapeutics, a comprehensive review of transforaminal lumbar injections is provided. Patient preparation, special considerations, procedural technique, complications, and alternative approaches are discussed.
  • ||||||||||  Cobenfy (xanomeline/trospium) / BMS
    Journal:  Schizophrenia - new treatments soon. (Pubmed Central) -  Nov 15, 2024   
    Recent clinical trials have shown that agonists of muscarinic acetylcholinergic receptors can improve both psychotic and negative symptoms in schizophrenia. Here, this new approach to the treatment of schizophrenia is reviewed in anticipation of the drugs being licensed clinically.
  • ||||||||||  cytarabine / Generic mfg.
    Journal:  Isocitrate dehydrogenase 2 mutation promotes cytarabine resistance in acute myeloid leukemia by Warburg effect. (Pubmed Central) -  Nov 15, 2024   
    To investigate cellular responses, the glycolytic inhibitor 2-deoxy-D-glucose (2-DG) was administered to the cells...The increase in glycolysis levels following IDH2 mutation may contribute to the reduced efficacy of Enasidenib in inhibiting the proliferation of IDH-mutant AML cells...BEZ235 significantly inhibits the expression of phosphorylated PI3K (p-PI3K), phosphorylated Akt (p-Akt), mTOR, glycolytic metabolism, and Ara-C resistance both in vitro and in vivo. Overexpression and mutation of IDH2 coordinate with the Warburg effect through the PI3K/Akt/mTOR pathway to promote Ara-C resistance in AML.
  • ||||||||||  Zolgensma (onasemnogene abeparvovec-xioi) / Novartis, Evrysdi (risdiplam) / SMA Foundation, PTC Therap, Roche, Spinraza (nusinersen) / Biogen
    Retrospective data, Journal:  Outcomes of early-treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study. (Pubmed Central) -  Nov 13, 2024   
    Early treatment of infants with SMA can improve outcomes as indicated by our cohort, all of whom sat independently and are without permanent ventilation. However, our study demonstrates ongoing disability in most children with two SMN2 copies despite early monotherapy and emphasizes the need for additional research, including earlier monotherapy, initial combination therapy, prenatal treatment, and non-SMN modifying treatments.
  • ||||||||||  Review, Journal:  Safety considerations for drugs newly approved for treating acute myeloid leukemia. (Pubmed Central) -  Nov 13, 2024   
    Since 2017, nine molecules were registered for newly-diagnosed AML: midostaurin, gilteritinib, quizartinib, enasidenib, ivosidenib, gemtuzumab ozogamicin, CPX-351, glasdegib, and venetoclax...However, in addition to their well-known cytotoxic activity, new molecules cause several unique, off-target toxicities. Also, potential drug-drug interactions (DDI) should be taken into consideration when choosing optimal first-line therapy; this remains a challenge in clinical practice.