MitoAction的动态

How can we work together to reduce the burden of #malaria and other diseases? This is one of the four pillars of MMV’s 2024–2030 strategy, and the one that can best leverage our 25 years of experience in drug discovery to the benefit of the wider global health community.? ? In this video, MMV’s Senior Director of Drug Discovery, James Duffy and H3D Foundation CEO Susan Winks go beyond the buzzwords to present a vision of how we can embrace new tools and ways of thinking to make gains against all diseases affecting vulnerable populations.?Watch it here: bit.ly/3WnqU8j?

Short but powerful video. Please watch

Turning Setbacks into Progress for Rare Disease Families For many families, navigating the rare disease journey has been riddled with delays and disappointments. We are changing that narrative now. Transforming the rare disease treatment landscape, making it faster and more efficient while keeping all the data in the hands of the client and moving them towards better health is our focus. We understand the frustration of working with multiple organizations that can't seem to provide the solutions you need. That's where Everlum Bio steps in. We're laser-focused on accelerating rare disease research and guiding families like yours through the disrupting process. Our specialized expertise and technologies have given us the ability to uncover insights and multiple potential therapies much faster than traditional approaches you may have previously encountered. Don't let delays define your rare disease journey any longer. Partner with Everlum Bio and experience the difference our solutions can make. Contact us today to learn what we can provide in the breakthrough you've been searching for. #PrecisionMedicine #RareDiseaseResearch #PersonalizedMedicine #BiotechLeaders #EverlumBio

Did you know that around 7,000 rare diseases affect countless individuals worldwide? Despite impacting so many, these challenges often remain unseen and under-researched. Today, let's change that. Let's break the silence around rare diseases and amplify the voices of those bravely fighting them. Share your thoughts, experiences, or even questions in the comments below. Together, we can raise awareness and support those navigating these challenges. Nikhil Reddy M.?Bharat Reddy?Arun Prasad M S Jayapala Reddy A V, PhD?Giridhar rao Pulla venkata?Kannan I #raredisease #researchforbettermedicine #research #msn #msngroup

Visit my wonderful colleagues as they network and discuss the challenges of rare disease! #clinicaltrials #clinicaldata #biometrics #biostatistics

Think you know rare disease recruitment?? Join Nadia Bodkin (RAM, Rare360) and Heather Flaherty (Level 5 Insights) at the Intellus Institute to hear fresh perspectives and test your skills with a fun roundtable exercise. Establishing the Gold Standard for Rare Disease Market Research Recruitment?? September 11, 2024 at 3:30 pm #raredisease #marketresearch #intellusworldwide #level5insights #levelupmarketresearch

One of the challenges of measuring the true impact of rare disease is the lack of appropriate quality of life assessment tools that adequately capture the impact of a condition, and its associated management, on an individual. We welcome work such as this from Truninger et al that seeks to develop better approaches to recording the impact of Pompe on children and adolescents affected by this condition. The PompeQoL questionnaire: Development and validation of a new measure for children and adolescents with Pompe disease Moritz Ilan Truninger,?Helene Werner,?Markus Andreas Landolt,?Andreas Hahn,?Julia Hennermann,?Florian Lagler, Prof. Dr. med.,?Dorothea M?slinger,?Charlotte Pfrimmer,?Rohrbach Marianne,?and Martina Huemer https://lnkd.in/eUecVDXt #pompe #raredisease #PROMs

Are you at Clinical Trials in Rare Diseases? Make sure to stop by the Greenphire booth to meet the team and learn how Greenphire can streamline your global #ClinicalTrial operations. Learn more about Greenphire today: https://www.greenphire.com #GOWithGreenphire #GreenphireMeansGO

Truly thanksful for the insighful discussions with all the inspiring persons I met EURORDIS-Rare Diseases Europe #ecrd2024 and i wanted to wrap up critical thoughts ??! Rare diseases face specific challenges in collecting, sharing, and making sense of scarce data. Treatment decisions are often hindered by cross-border political coordination and yet, #approval does not guarantee #affordability of the treatment for the patient, thereby creating further #inequalities. Fair decisions can only emerge from a rational #decisionmaking process, which begins with full #datasharing and a complete description of the assessment fostering #transparency. We propose a solution that accounts for #multipleoutcomes simultaneously, especially their correlation, to rank #priorities effectively. This framework can assist in the debate among patients, clinicians, and payers and provide empirical evidence that can be #reproducible in every country. If you want to move decisions in rare diseases forward, please contact me ? ? ??

Today, Feburary 29, marks ?????????????????????????? ???????? ?????????????? ??????, a time to shine a spotlight on the 300 million individuals worldwide living with rare diseases, along with their families and caregivers. Rare diseases may be uncommon individually, but their collective impact is profound. At Julius Clinical, we recognize the importance of raising awareness and fostering change for those facing unique medical challenges. By uniting in advocacy and support, we can make strides towards better understanding, improved treatments, and enhanced quality of life for all affected by rare diseases. #RareDiseaseDay