Lumanity

Duchenne’s Muscular Dystrophy (DMD) and Beckers Muscular Dystrophy (BeMD) have a global prevalence of 0.0048% and 0.001%, respectively. Due to the rarity of these conditions, and the multi-disciplinary nature of care management, identifying these patients in real world data (RWD) can be challenging. Uncertainties in the long-term safety of novel treatments for DMD/BeMD, such as cell and gene therapies, further increase demand for robust, longitudinal real world data within these disease populations. The PHARMO Institute, part of Lumanity, delivers research insights into DMD and BeMD and the PHARMO Data Network offers great research potential for these rare disease populations: • 20+ years of follow up across multiple patient healthcare settings • ~199 patients identified with DMD and ~245 with BeMD diagnosis within the General Practitioner (GP) database between 2003-2023 • Clinical laboratory data to identify the target cohort via creatine kinase concentration testing and deeper insights into the genetic makeup of patients via the pathology registry Our RWD is available to address a wide range of use cases from treatment patterns, disease progression, safety, and validation of surrogate endpoints of your trial. We invite you to put our data to the test. Learn more about our expertise at https://buff.ly/4eMA5Fs or email us at contact@lumanity.com to discuss your real world evidence research needs. #raredisease #rarediseases #duchennes #duchennesmusculardystrophy #dmd #beckers #beckersmusculardystrophy #bmd #PHARMO #RWD #RWE #realworlddata #realworldevidence #SIGMAconsortium #ENCEPP #outcomesresearch #safetystudies #surrogateoutcomes #longitudinaldata

Will you be at next week's Cell Therapy for Autoimmune Disease Summit? If so, make sure you stop by our exhibit booth and schedule time to connect with our experts on-site, including Kristin Abramo, Ph.D., David Lomb, and Danielle Marra. Reach out to our colleagues directly or email contact@lumanity.com to find time to connect during the summit. Learn more at https://buff.ly/3Vw9qpN #CellTherapy #AutoimmuneDisease #BioMarkers #ClinicalDevelopment

Alexander Fink and Ed Saltzman sat down together at the recent BIO-Europe conference and discussed why biopharma must better prepare for commercialization, even if the aim is to seek partnerships. In the video, they tackle common myths surrounding this topic and offer practical guidance on how teams can effectively approach the commercial thinking that is essential for success. Watch the full video at https://lnkd.in/ePJzByNd and be sure to read our white paper on the topic at https://lnkd.in/exUhg7rr #biopharma #biotech #commercialization #commercializationpartnerships #commercialsuccess #BIOEurope

We are counting down to the upcoming Pharmaceutical Launch Excellence Summit. Be sure to reach out to contact@lumanity.com or directly with our colleagues to connect with our experts, Keitel Suri and Jeff Hart. For more information about the summit, visit https://buff.ly/4esJP7r #LaunchExcellence #Biopharma #ClinicalDevelopment #CommercialLaunch #PharmaceuticalLaunch

We're just a couple weeks away from the upcoming 13th Annual Life Science Strategic Patient Advocacy Conference. Be sure to reach out to contact@lumanity.com to connect with Holli Kawadler, PhD who will be on-site in Philadelphia. For more information about the summit, visit https://buff.ly/3YJqadA #PatientAdvocacy #PatientEngagement #LifeSciences #PatientAdvocate

In this third episode of our podcast miniseries on integrated evidence generation planning (IEGP), our experts discuss key regional considerations when forming a strategy to address specific needs and challenges across diverse geographies, populations and health systems. Listen now: https://buff.ly/3AQ1PLo If you missed the first two episodes, make sure to check them out here: Episode 1: https://buff.ly/3Z0Pps4 Episode 2: https://buff.ly/3Zdcut4 #IEGP #IntegratedEvidenceGenerationPlanning #MAPS #MedicalAffairs #MedAffairs #PodcastSeries #PodcastMiniseries #PodcastEpisode #Pharma #PharmaStrategy

Our health economics, outcomes research, and real world evidence experts are on-site in Barcelona for ISPOR Europe. If you haven't yet, be sure to stop by booth 921 to catch up with our colleagues, grab some swag to take home, and participate in a short questionnaire at the stand! Learn more about our presentations and who is at the conference at https://buff.ly/3V0GOo2 #ISPOREurope #EUHTA #HEOR #HealthEconomics #OutcomesResearch #PatientCenteredOutcomes #RWE #RealWorldEvidence #ISPOR ISPOREU

In this article for QT9 QMS, Menaka Manokaran, Associate Director of Regulatory Affairs at Lumanity, reviews the FDA approval process for medical devices. The article examines Special 510(k) and Abbreviated 510(k) submissions, as well as premarket approval, the most stringent type of device marketing application required by the FDA. Read the article on the QT9 QMS site: https://buff.ly/4fLDaGx #QT9QMS #ClinicalAndRegulatory #FDA #MedicalDevices #MedicalDevice #FDAApproval #Regulatory #RegulatoryAffairs

In case you missed it, we recently discussed the use of real world data to guide precision oncology drug development. The discussion featured the topics of: - Strategic challenges in precision oncology - Approaches for navigating these uncertainties - The role of real world data and analytics and whether the use of real world evidence can be optimized to close gaps in target identification/biomarker discovery, indication prioritization, and disease & market understanding. Watch the recording of the webinar at https://buff.ly/3AV03bP #RWE #RWD #Oncology #DrugDevelopment #PrecisionOncology #RealWorldEvidence #RealWorldData #Biomarket #Cancer

A formidable group of experts are at #ISPOREurope this week! If you're in Barcelona for the conference, be sure to visit booth 921 and catch up with all of our colleagues on-site. For the full list of our presentations and experts in attendance, visit https://lnkd.in/eRTiSqhK #HEOR #HTA #HealthEconomics #OutcomesResearch #PatientCenteredResearch #ISPOR