4D Molecular Therapeutics’ Post

Speeding up #clinicaltrials for #rarediseases is now a necessity. Because the people affected with the rare conditions are still waiting for innovative therapies that can change their lives. Check out how our offerings can help fast-track rare disease studies: https://bit.ly/3UOqFmq #ClinicalResearch #LifeSciences #RareDiseaseDay2024

TAPS is Twin Anemia Polycythemia Sequence, a rare disease affecting 3-5% of monochorionic twins. It's time to start raising awareness of TAPS, and changing guidelines to save lives. Learn more about TAPS here: https://bit.ly/32AXytG #tapssupport #tapsisreal

Today is Rare Disease Day! A special day to raise awareness around the world, improve access to treatment and support patients and families 💜 What are rare diseases? All of those that affect less than 1 in 2000 people, it is estimated that 300 million people live with a rare disease worldwide 🌎 The principal challenges for patients living with rare diseases are the lack of information, which leads to a delay in the diagnosis, and the limited access to treatment and care How can we help? Get involved in Rare Disease Day activities, spread the word, and share knowledge 💚 Unite for change! Unite for equity! #RareDiseaseDay #LightUpForRare

Today February 29th, the rarest day of the year, is Rare Disease Day! Rare Disease Day is a global movement raising awareness for those affected by rare diseases like my daughter who has #FoxG1Syndrome. Those with rare diseases often receive delayed diagnosis or misdiagnosis and research is typically underfunded. Doing my small part in raising awareness to work towards equity in social opportunity, healthcare, and access to diagnosis and therapies for the many living with rare diseases. #rarediseaseday #rarediseaseday2024 #lightupforrare #geneticdisorders

Today, on #RareDiseaseDay, we are reminded of the importance of amplifying the voices of over 350 million individuals worldwide affected by one of 7,000 rare diseases (1,2). To provide some perspective, if this community formed a country, it would rank as the third most populous globally after India and China. Throughout my career, I've had the opportunity to meet families and individuals affected by these conditions, gaining firsthand insight into the day-to-day challenges they face and the transformative impact of certain treatments. It's particularly heartbreaking that 80% of these conditions are hereditary, often afflicting children. These encounters underscore the human element driving our efforts to develop and manufacture therapies. However, achieving equitable access to diagnosis and access to treatment remains a significant challenge. This Rare Disease Day, I stand with CSL in reaffirming our commitment to patients and caregivers, holding ourselves accountable as partners in addressing these challenges. One crucial issue is the need for early diagnosis; it’s disheartening that it currently can take an average of 5 years to receive a proper diagnosis (3). Additionally, another area requiring attention is equitable access to treatments. While some progress has been made, with initiatives like the EU Orphan Regulation bolstering advancements, it's essential to note that 95% of rare diseases still lack approved treatments - underscoring the urgent need for research incentives. This prompts our active efforts to raise awareness and collaborate with stakeholders so that everyone receives prompt diagnosis and access to timely treatment. As I reflect on the ongoing revision of the EU pharmaceutical package, I see an opportunity to foster innovation, improve access to treatments and enhance supply sustainability within Europe. For me, it's about making sure that every patient, especially those with rare and complex diseases, can have access to the innovative and safe medicines they need. As we listen to the stories of patients, caregivers, and healthcare workers this Rare Disease Day, let's reflect on how we can further advocate for this community. These narratives remind us of the urgent need for policies that prioritize patient access to innovative treatments. I invite you to share in the comments how these stories have inspired your priorities for improving access for all. #RDD2024, #EUhealthcare #access #csl References: 1. National Organizations for Rare Disorders (NORD) https://lnkd.in/dJZ3-R_y 2. National Human Genome Research Institute https://lnkd.in/dZVM6ybB 3. Global Genes https://lnkd.in/dE-xJ5AE

May is #LymeDisease Awareness Month, and at T2 Biosystems, we're dedicated to shedding light on this prevalent but often misunderstood illness. Lyme disease affects millions worldwide, yet diagnosis can be challenging due to nonspecific symptoms and unreliable tests. Join us in raising awareness this month and learn how the #T2Lyme Panel has the potential to make a difference in the fight against Lyme disease. #HealthInnovation https://ow.ly/fbpM50RikUE

🩷💚💙 Today is Rare disease day ! On this Rare Disease Day, let's shine a spotlight on those facing the challenges of neuromuscular diseases. Your resilience in the face of adversity is truly inspiring. Together, let's raise awareness, support research, and foster a community of understanding and empathy. Every voice matters, and every step taken towards better understanding and treatment brings hope to those affected. Let's stand together, today and every day, in solidarity with those battling neuromuscular conditions. You are not alone. #RareDiseaseDay #NeuromuscularAwareness #ERNs #NMD EURORDIS-Rare Diseases Europe

Today is the first day in my initiative to raise awareness for Rare Disease Day, coming up on the 29th February. I will post something about one rare disease every day during February. Mostly, these will be personal stories from people living with a rare disease. The stories that are coming, will truly humble you. Amazing people with resilience beyond my comprehension! But...today is a bit different. Today is von Willebrand awareness day, so that seemed like the obvious place to start this initiative. There is a great initiative being run by the European Haemophilia Consortium. I have written an introduction to VWD, and the reasons for its under diagnosis. Maybe some of the symptoms will resonate with someone, as around 1% of the population have this condition, and many will not have been yet diagnosed. There are some useful links at the bottom of the article, where you can find more information. #rarediseaseawareness #bleedingdisorders

 Rare Disease Day serves as a powerful platform to celebrate progress whether through improved treatments, early interventions, or increased awareness which renews hope for a brighter future for those suffered from rare diseases.” “Being part of Biopharma-mea Access Team, I witness the challenges faced by individuals with rare diseases. My empathy and compassion play a crucial role in understanding their unique needs and advocating for better access to treatments, medications, and support. Biopharma-mea #RareDiseaseDay #ShareYourColours #RareDisease.

In recognition of Myasthenia Gravis Awareness Month this June, we are dedicated to shedding light on this challenging condition. Myasthenia Gravis affects between 140 and 361 individuals per million globally and approximately 1,500 individuals in the GCC region (Salari et al., 2021). This chronic autoimmune neuromuscular disease leads to muscle weakness, significantly impacting patients’ daily activities. In severe cases, it can affect essential functions, including the ability to breathe normally. An estimated 15% to 20% of MG patients experience myasthenic crises during their lifetime, with women being affected twice as often as men (Huang et al., 2021). Furthermore, nearly one-fifth of patients admitted for myasthenic crises require ongoing care through rehabilitation centers (Köhler et al., 2011). By raising awareness and understanding of Myasthenia Gravis, we can ensure that those affected receive the support and treatment they need. Join us this month in spreading the word and making a difference. References: Salari N, et al. Global prevalence of Myasthenia Gravis and the effect of geographic location and measurement type: a comprehensive systematic review and meta-analysis. J Transl Med. 2021 Dec;19(1):516. Huang Y, et al. Epidemiological and clinical characteristics of patients with Myasthenia Gravis in China: a systematic review. Front Neurol. 2021;12:767961. Köhler W, et al. Myasthenic crisis: current strategies and new perspectives. J Clin Apheresis. 2011;26(6):347–55.