Prader-Willi Syndrome Association | USA’s Post

Bringing a new therapy to patients with rare diseases isn’t easy, but it can be life-changing. 🔬 In our latest webinar, former Reata Pharmaceuticals, Inc. CEO Warren Huff shares the story of Skyclarys—the first FDA-approved treatment for Friedrich’s ataxia. Key highlights include the use of natural history studies and the importance of patient advocate collaboration. ✨ Read the summary and watch the webinar in full 👉 https://lnkd.in/gA7-egDf #RareDiseases #FriedrichsAtaxia #DrugDevelopment

Sjogren’s Syndrome Pipeline 2024: Clinical Trials Assessment, FDA Approvals, Therapies, and Key Companies Involved by DelveInsight | Novartis, Johnson & Johnson, Dompe Farmaceutici, Sylentis, Amgen: (Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, Sjogren’s Syndrome pipeline constitutes 20+ key companies continuously working towards developing 22+ Sjogren’s Syndrome treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes … Continue reading → #Business #HealthMedicine #MarketingSales #PharmaceuticalsBiotech

NImmune Biopharma, Inc’s Founder, Executive Chairman & CEO Josep B. was recently honored as a Virginia Success Story at THRiVE, Virginia Bio’s largest conference of the year. Dr. Bassaganya-Riera described his role in building a thriving biotech innovation ecosystem from the ground up, encompassing NImmune Biopharma, The NIMML Institute, and BioTherapeutics Inc., alongside Landos Biopharma, Inc., which was recently acquired by AbbVie. He underscored the groundbreaking potential of NImmune’s therapeutics in addressing the unmet clinical needs of millions of patients suffering from autoimmune diseases worldwide. NImmune’s lead drug, omilancor, is a first-in-class, best-in-class, oral, once-daily therapeutic in Phase 3 clinical development with a planned NDA filing for Ulcerative Colitis by 2026 and in Phase 2 clinical development for Crohn’s disease. Additionally, NIM-1324, a first-in-class, oral, once-daily, systemically distributed LANCL2 agonist, is in Phase 2 clinical development for the treatment of systemic lupus erythematosus, lupus nephritis, rheumatoid arthritis and other undisclosed indications. Watch Dr. Bassaganya-Riera’s full remarks below.   #VirginiaBio #THRiVE #PrecisionMedicine #LANCL2

The Week that was in Medical Cannabis IMPORTANT NEGATIVE TRIALS JUST PUBLISHED - cancer - COPD - driving Cannabinol oil or placebo in advanced cancer-disease progression and survival: a secondary analysis. O’Leary C et al. BMJ Support Palliat Care. 2024 Feb 2:spcare-2023-004746. doi: 10.1136/spcare-2023-004746 https://lnkd.in/gazsY-ar  Effects of Dronabinol on Dyspnea and Quality of Life in Patients with COPD. Zaid A et al. Chronic Obstr Pulm Dis. 2024 Feb 2. doi: 10.15326/jcopdf.2023.0401 https://lnkd.in/gJzF8Y36 A semi-naturalistic open-label study examining the effect of prescribed medical cannabis use on simulated driving performance. Manning B et al and Thomas Arkell. J Psychopharmacol. 2024 Feb 8:2698811241229524. doi: 10.1177/02698811241229524. https://lnkd.in/gER9Mb84 #theweekthatwasinmedicalcannabis #medicalcannabis #medicinalcannabis

AbbVie’s $8.7 Billion Cerevel Buy Starts to Pay Off with Phase III Parkinson’s Win AbbVie has announced that its investigational Parkinson’s disease drug tavapadon met its primary efficacy endpoint in the Phase III TEMPO-1 trial, showing significant improvements in motor and non-motor symptoms in patients with early-stage disease. Tavapadon was originally developed by Cerevel Therapeutics, which AbbVie acquired for $8.7 billion in December 2023. The companies formally completed the deal last month. AbbVie is also running the TEMPO-4 trial, which will assess the long-term safety and tolerability of tavapadon, and the TEMPO-2 study, which is looking at a flexible-dose regimen of tavapadon monotherapy. Topline data from the latter study are set to come out by the end of 2024. https://lnkd.in/ghki4BsY #aspenalert #biotech #bioprocess

So glad to see more concrete guidance coming out of the FDA for #DiversityActionPlans. Take time to read the 26pg update --- and let's set up some more channels for dialog to get some feedback back to the agency! What's fascinating is that this comes at a time when recent analysis shows that "Prior to 2020 no drugs had a PMR/C to further characterize use in a more representative population, and in the last three years, more than half of novel oncology approvals have had such a PMR/C (21/40, 53%)." https://lnkd.in/gNczUwNA #ReachFurther #RepresentationMatters #DiversityInClinicalTrials #BuildingBridges #CommunityEngagement #FDA

With the approval of OGSIVEO™ (nirogacestat), patients must contact their healthcare provider to see if the drug is right for them. SpringWorks Therapeutics, the pharmaceutical company that developed the drug, has several resources available to desmoid tumor patients. ✔️ SpringWorks Therapeutics has a patient support services program called SpringWorks CareConnections™ for people prescribed OGSIVEO™ (nirogacestat). Physicians and patients can contact 1-844-CARES-55 (1-844-227-3755) or visit springworkstxcares.com. This is a free, personalized patient support program to help navigate insurance coverage, answer questions about treatment, and help patients start and stay on track with OGSIVEO™ (nirogacestat). ✔️ Official website: Ogsiveo.com ✔️ Official Facebook page: facebook.com/OGSIVEO ✔️ Official Facebook group for health care providers: facebook.com/ogsiveoHCP #Desmoid #DesmoidTumors #DesmoidTumor #fibromatosis #aggressivefibromatosis #DTRF #DesmoidTumorResearchFoundation #Desmoidian #desmoidresearch #FAP #familialadenomatouspolyposis #OrphanDisease #raredisease #oncology #research #hope #OGSIVEO #nirogacestat #expandedaccess

Milestone Achieved: FDA Greenlights Gilead's Newly Poised “Livdelzi” to Make a Big Impact Perhaps you caught our previous LinkedIn post that was shared by us 2 months ago. As it happens, what we’ve been hoping for has come to fruition. Gilead Sciences has now received FDA approval for Livdelzi (seladelpar), a new treatment for a rare liver disease known as primary biliary cholangitis. This approval is crucial for patients who can’t tolerate or don't respond well to existing treatments. Livdelzi is now available alone or in combination with other therapies and has strong potential in the market. How will Livdelzi's FDA approval reshape the options available for patients with primary biliary cholangitis? RBC Capital Markets analyst Brian Abrahams noted that Livdelzi's "cleaner label and itch relief benefits" are likely to give it a competitive advantage. Feel free to check out the article linked in the comments below.   #Livdelzi #FDAApproval #PrimaryBiliaryCholangitis #GileadSciences #Biotech #Pharma #HealthcareInnovation #LiverDisease #NewTreatment #CymaBayTherapeutics #DrugDevelopment #MarketImpact #MedicalBreakthrough

The Endocannabinoid System and Colitis: Exploring the clinical data surrounding the use of cannabis for inflammatory bowel disease Preclinical data supports the use of cannabinoids for gastrointestinal inflammation, but does this translate into patient outcomes? Part 1 of this series on cannabis for colitis outlined the preclinical data which evaluated the use of cannabis for inflammation of the GI tract. Although the preclinical data largely supports the use of cannabis in combating inflammation, part 2 of this blog will continue to explore cannabis in the context of colitis and inflammatory bowel disease (IBD). This second part of our series transitions from the preclinical insights discussed earlier to an examination of existing clinical data. Read more here: https://lnkd.in/eDTAAe2P

Redx Pharma PLC (AIM:REDX) said it has kicked off a phase I clinical trial for RXC008, a 'first-in-class' ROCK inhibitor treatment targeting fibrostenotic Crohn's disease. This study aims to assess the safety and pharmacokinetic profile of the drug - in other words, its activity in the body over some time, including the processes by which RXC008 is absorbed, distributed in the body, localised in the tissues, and excreted. It will be assessed in healthy volunteers with the results from the study expected by the end of this year. Chief executive Lisa Anson told investors: "We are delighted to confirm that the first participant has been dosed in the RXC008 phase I clinical study. "RXC008 is a potential first-in-class treatment for patients with fibrostenotic Crohn's disease, a debilitating condition where successive surgeries are the only treatment option available today. More at #Proactive #ProactiveInvestors #AIM #REDX #Crohnsdisease #treatment http://ow.ly/SBp8105k03L