Madrigal Pharmaceuticals’ Post

We are celebrating 20 years of contributions to therapeutics for metabolic diseases, and we couldn't have done it without YOU. To all our incredible team members, study participants, clients, patient advocacy organizations, and the scientific community at large – THANK YOU for your support and dedication. Your collective efforts have aided in our success and shaped the impactful contributions we've made over the years. We remain steadfast in our commitment to our mission – finding better treatments for #diabetes, #obesity, #MASLD / #MASH, and related diseases across the metabolic continuum. https://bit.ly/3KNlRsG #MetabolicResearch #DiabetesResearch #ObesityResearch #MASLDResearch #MASHResearch #NASHResearch #NASH #NAFLDResearch #NAFLD #AnniversaryCelebration #MetabolicContinuum

In a remarkable decision posted earlier this month, INESSS stands alone in failing to recognize the therapeutic value of XPOVIO. This despite Health Canada approval, a positive CADTH decision, Quebec clinician and patient support and positive funding recommendations from such organizations as Australia's PBAC and most recently Britain's NICE. This negative recommendation by INESSS raises many concerning questions but most importantly isolates the Quebec patients and treating community on a very distinct and unique island. We see, yet again, the 'postal code lottery" of access at play in our country. We have to find better, more consistent solutions for Canadian patients. Read on our website (https://lnkd.in/gQB72-i8) and we refer you to FORUS press release online links dated April 24th for additional information. FORUS press release on April 24th, 2024 in French. (https://lnkd.in/gPkdC3FP) FORUS press release on April 24th, 2024 in English. (https://lnkd.in/gM7usdyR) #multiplemyeloma #bloodcancer #science #patients #canada #funding

🌍 Today, on #RareDiseaseDay, at Oryzon we want to show our support and raise awareness for the 300 million people living with rare disease around the world and their families and carers.   🧬As a company whose mission is to improve people's quality of life through new and innovative treatments, we understand the unique challenges faced by patients and their families when it comes to accessing effective treatments for these uncommon conditions. That's why we remain committed to researching and developing innovative therapies for diseases with significant unmet medical needs both in oncology and CNS.   We also recognize the importance of advocating for equitable access to diagnosis, treatment and health and social care for people affected by a rare disease, which is the central motif of this day.   💊🔬 Our focus on R&D is at the core of our mission to improve the lives of patients and bring hope to those who need it most. Together, we can make a difference and ensure that no one is left behind. #Oryzon #RareDiseaseDay #research #epigenetics #biopharma  

Today, kicks off our 4th annual Lupus Clinical Investigators Network (LuCIN) Community Meeting, hosted by #LupusTherapeutics, the clinical affiliate of the Lupus Research Alliance. This annual meeting brings people with #lupus together with thought leaders, clinical investigators, study coordinators, research & industry partners to discuss the progress and advancements being made in #LupusClinicalResearch and treatment, and the importance of health equity and access in #lupus clinical trials. Learn more about LuCIN at https://bit.ly/LuCIN2024 #LuCINCommunity #LupusAwarenessMonth

New challenges and new opportunities for ANCA-associated vasculitis. We took stock the situation about the unmet needs in AAV from clinicians’ and patients’ perspective. https://lnkd.in/dwf-JmqJ

No treatment for Lp(a) cholesterol even though it is one of the biggest genetically determined risk factor of #cardiovascular disease. Huge unmet need The #siRNA therapy lepodisiran trial is advancing to phase 3 ACCLAIM-Lp(a) trial, evaluating both secondary and high-risk primary prevention to treat high lipoprotein(a) (Lp(a)) levels for cardiovascular disease https://lnkd.in/eXmWrnPB

🌐UPNAT recently participated in the inaugural meeting of the UK Rare Disease Research Platform. This initiative, backed by a £14 million investment over five years from the Medical Research Council, part of UK Research and Innovation, and the NIHR (National Institute for Health and Care Research), aims to enhance the diagnosis and treatment of rare diseases by leveraging the collective expertise of the UK's research community.     🧬 Dedicated to addressing diseases affecting fewer than one in 2,000 people, this platform comprises a central hub and 11 specialised nodes across the UK, each focusing on distinct rare disease areas.     🤝 NATA is proud to be part of UPNAT (UK Platform for Nucleic Acid Therapeutics), a collaboration with UCL and the University of Oxford developing a translational and regulatory road map for nucleic acid therapies for rare diseases.    We look forward to contributing to this transformative platform and working collaboratively to address the shared challenges in rare disease research.      #RareDisease #UPNAT #Collaboration #Innovation #NATA #oligonucleotides #RNAtherapeutics #RNAtherapies #AdvancedTherapies #NATs #Healthcare #Impact #Industry #clinicalresearch #health #Medicine 

The Innovation and Value Initiative (IVI) and EveryLife Foundation for Rare Diseases have partnered to identify common patient-centered outcomes and achieve a consensus on addressing unique research challenges. We hope these findings develop effective research approaches for patient engagement and recommendations for patient-centered outcomes, leading to informed outcome selection that is important to patients, caregivers, and families. Read the report: https://lnkd.in/g5UP--5g 

C-Path is thrilled to announce a significant addition to its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®). CHOC Children's has generously contributed clinical trial data related to mucopolysaccharidosis, reinforcing the power of collaboration in advancing rare disease research. In light of CHOC’s collaboration with RDCA-DAP, the Critical Path for Lysosomal Diseases Consortium, part of C-Path’s efforts, stands to benefit from this partnership. CPLD, which is committed to uniting industry leaders, researchers, regulators, and patient advocacy groups, aims to advance diagnostic and therapeutic approaches for lysosomal diseases. Kanwaljit Singh, MD, MPH, Executive Director of CPLD, commended this effort, stating, “CHOC’s data sharing with RDCA-DAP enhances our pursuit of new treatments and underscores the impact of collaborative innovation in rare disease research.” Full details, here: https://lnkd.in/g3JTxNM6 Collin Hovinga Alexandre Bétourné, PhD, PharmD, PMP #CPath #CHOC #RDCADAP #raredisease #clinicaltrial #datasharing #drugdevelopment #globalhealth #collaboration