Keck School of Medicine of the University of Southern California’s Post

University of Miami Health System and Holtz Children’s Hospital Physicians Evaluated the Effectiveness of ELEVIDYS University of Miami Health System and Holtz Children’s Hospital physicians are playing a major role in getting ELEVIDYS access. The therapy delivers a functional micro-gene to muscle cells, potentially arresting DMD progression and, in some cases, possibly reversing it. Raniya was the first female ever treated with ELEVIDYS. Siblings Raniya, who is 15 years old, and Amir, 8, received their single ELEVIDYS infusions at Holtz Children’s Hospital in late October. They may have to wait several months to learn if the treatment was successful, but early signs are encouraging. “At her recent appointment, Raniya was already doing better, feeling stronger, walking more and not relying on a wheelchair as much,” said Dr. Lopez-Alberola. Read More: https://lnkd.in/dmby8RMW #dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna #elevidys #sarepta

My talk at the National Institute on Aging (NIA) workshop on dementia with Lewy bodies (DLB): https://lnkd.in/eTuah747. Workshop was on advances in understanding translational and clinical aspects of DLB. So, my talk is on what we learned from our clinical studies that informs on DLB pathogenesis and designing clinical trials in DLB. But, also a very good summary of everything we know about our drug, neflamapimod, and its potential to treat underlying disease process in DLB.

📈 Patients of physicians with higher adenoma detection rates during colonoscopy often have a lower risk of #colorectal cancer after screening #colonoscopy. 🔎 A recent observational study sought to determine if there is a correlation between the improvement of ADR and lower rates of CRC in patients who undergo colonoscopy:

🫀 #HypertrophicCardiomyopathy is the most common genetic heart disease: it is estimated that approximately 1 in 500 individuals suffer from it.   ✅ Some time ago, #HCM was considered difficult to treat and had poor prognosis, but hanks to #research and #therapeutic and diagnostic #innovation, its approach is changing considerably.   🔬 At Bristol Myers Squibb we are driven by a single passion, to continue transforming the lives of #patients through our innovative #Science. That's why we are participating these days in Lisbon at the World Congress on Acute Heart Failure, organized by the European Society of Cardiology, where we share with the scientific community our latest advances for patients with #HCM.   🙌🏻 Only by working together can we achieve the greatest benefit for #patients! #HeartFailure2024 #HFAmotions #HFA_ESC #HFAsnacks #20yearsHFA #BMSIberia #BMSPortugal #BMSCommunity #BMSTransformingLives #WorkingTogetherforPatients #BMSTransformingPatientsLives 

Join Lisa Richards, NP from UC San Diego Health, as she breaks down a pivotal study from the A Phase 3, Randomized, Controlled Trial of Resmetirom in #NASH with Liver Fibrosis! Learn how this groundbreaking treatment is advancing clinical outcomes for patients at bit.ly/4j3nssO!

We’re thrilled to share more encouraging updates from Neurizon Therapeutics’ Open-Label Extension (OLE) Study in patients with ALS. The latest data reveals that treatment with NUZ-001 remains well-tolerated and continues to demonstrate promising results in slowing disease progression and extending life expectancy compared to untreated matched controls. Our commitment to advancing this innovative therapy remains stronger than ever as we work to bring hope and new possibilities to the ALS community.   📖 Read the full press release here: https://lnkd.in/e-e2ydzY #ALS #NeurodegenerativeDiseases #NeurizonTherapeutics #HorizonOfHope

Of the 98 #Nobel prizes in medicine, 79 have been innovations that included #preclinical disease models in the early discovery phase of research. MLM Medical Labs currently maintains a portfolio of 60+ in-vivo models to address a wide range of diseases including #fibrosis, #arthritis, #respiratory disease, #dermal conditions, #inflammatory disease, and #sepsis. Our scientists also work with Sponsors to customize existing or innovate new models to meet specific research goals. Learn more about our in-vivo models >> https://lnkd.in/gNv3Cb2m #preclinicalresearch #translationalresearch #preclinicaldiseasemodels #diseasemodels

Of the 98 #Nobel prizes in medicine, 79 have been innovations that included #preclinical disease models in the early discovery phase of research.  MLM Medical Labs currently maintains a portfolio of 60+ in-vivo models to address a wide range of diseases including #fibrosis, #arthritis, #respiratory disease, #dermal conditions, #inflammatory disease, and #sepsis. Our scientists also work with Sponsors to customize existing or innovate new models to meet specific research goals. Learn more about our in-vivo models >> https://lnkd.in/gNv3Cb2m #preclinicalresearch #translationalresearch #preclinicaldiseasemodels

LATEST NEWS! VECTORS study first participants randomized in Crohn’s disease and transmural healing by using intestinal ultrasound (IUS). We're pleased to confirm the first participants have been randomized in the VECTORS study! VECTORS is a unique study in #CrohnsDisease (CD) research aiming to answer a key clinical question related to the optimal treatment algorithm to achieve a target of transmural healing in Crohn’s disease. VECTORS will also provide key insights into the use of intestinal ultrasound as a non-invasive modality in Crohn’s disease clinical trials and clinical practice. VECTORS is a collaborative study between Alimentiv and Takeda and a testament to the dedication of both organizations to improve the lives of patients living with IBD. Learn more: https://bit.ly/3zkz1tX #Alimentiv #IBD #Ultrasound #MedicalResearch #ClinicalTrials