ICT Spring 2024 is now live! ☀️ Featuring insights in Decentralised Clinical Trials, Orphan Drug and Rare Disease Trials, and much more! Read here: https://bit.ly/3URlpxX #ClinicalTrials #decentralisedclinicaltrials #rarediseases #orphandrugs #pharmaceuticalindustry
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ICT Spring 2024 is now live! ☀️ Featuring insights in Decentralised Clinical Trials, Orphan Drug and Rare Disease Trials, and much more! Read here: https://bit.ly/3URlpxX #ClinicalTrials #decentralisedclinicaltrials #rarediseases #orphandrugs #pharmaceuticalindustry
ICT Spring 2024 is now live! ☀️ Featuring insights in Decentralised Clinical Trials, Orphan Drug and Rare Disease Trials, and much more! Read here: https://bit.ly/3URlpxX #ClinicalTrials #decentralisedclinicaltrials #rarediseases #orphandrugs #pharmaceuticalindustry
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Following 2023’s successful EL-PFDD meeting, our new Voice of the Patient Report is a critical next step in the IPPF’s engagement with FDA and other decision-makers. As we’ve learned, there are significant obstacles facing #pemphigus and #pemphigoid patients’ access to potential future therapies. To overcome these obstacles, we must empower the voice of a strong coalition of patients, caregivers, researchers, and clinicians so that regulatory agencies and industry partners understand what truly matters to people living with these diseases.
To celebrate Rare Disease Day 2024, the IPPF is excited to announce the publication of Understanding the Unmet Needs of the Pemphigus & Pemphigoid Community, a summary of the 2023 patient-focused drug development meeting. Learn more at www.pemphigus.org/el-pfdd. #healourskin #RareDiseaseDay
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Celltrion's infliximab biosimilar, CT-P13, was found to be just as effective and safe as the original brand name drug. This means a potentially more affordable option for treating patients with Crohn disease! Click the link to read more about the results found in a retrospective, real-world study in Japan. https://lnkd.in/eXUZDwp5
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🔬 Today, on Rare Disease Day, we stand in solidarity with those affected by sarcoidosis. Did you know that, as reported in the Journal of Internal Medicine in March 2023, the annual incidence of sarcoidosis ranges from 1 to 15 per 100 000 depending on the region? 🌍 At #Molecure, we're committed to making a difference. Our leading drug candidate, #OATD01, is as a first-in-class dual chitinase inhibitor aimed at treating fibrotic and inflammatory diseases, including pulmonary interstitial diseases like sarcoidosis and idiopathic pulmonary fibrosis (IPF). OATD-01 has successfully passed pre-clinical and clinical Phase 1 studies, demonstrating strong anti-inflammatory and antifibrotic properties. Currently in Phase 2 clinical trials, that will be conducted in the United States and Europe, #OATD01 holds promise for those battling these challenging conditions 🧬 Together, let's raise awareness and support for all those impacted by rare diseases 🤝 #RareDiseaseDay #SarcoidosisAwareness
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Rare disease research faces a common challenge: how do you study new treatments and advance research when there aren’t a lot of people living with the rare disease? And how do you incentivize drug development sponsors to invest in these important studies and encourage patients to participate in clinical trials? #RareDisease #ClinicalTrials #PatientVoice
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Excited to share my article, Long-Term Safety of Guselkumab in Patients with Psoriatic Disease: An Integrated Analysis of Eleven Phase II/III Clinical Studies in Psoriasis and Psoriatic Arthritis, which was published in Drug Safety earlier today! https://lnkd.in/e4Vr7P-3
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Today marks the beginning of #HS Awareness Week. HS is a debilitating and often painful chronic inflammatory disease that does not have a cure. Diagnosis can be challenging, with it taking on average seven years to receive a formal diagnosis. Not only can a lengthy HS diagnosis lead to increased depression and anxiety, studies have also shown that diagnosis is key to preventing further irrevocable damage and scarring. This is why we’re championing awareness and advocating for further research, to help patients get the support they need and to slow further disease progression. At MC2 Therapeutics we are on track to file an IND in mid-2025 for a Phase 2b clinical trial for our first-in-class oral drug candidate MC2-32 in HS. This will be an instrumental moment as we spearhead our efforts to develop safe and effective treatments for patients with high unmet needs. Learn more here: https://lnkd.in/dbRsAh9Q
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Did you know that today is #RareDiseaseDay? Check out Certara Talks with our experts, Oxana Iliach, Rajesh Krishna, David Wesche, and Paul Martin, who discuss the combined efforts and expertise needed for a successful rare disease drug submission. They also share insights into the regulatory challenges that rare disease development programs need to think about. ▶️ Watch now: https://ow.ly/CkGc50QH9HZ Curious to delve further into the world of rare disease drug development? Explore more at: https://lnkd.in/eTDeSvhH #RareDiseaseDay #DrugDevelopment #CertaraTalks
Rare Disease Day 2024
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"Bridging the Gap, Empowering Patients: A Collaborative Approach to Rare Disease Research" underscores the power of collaboration and patient-centered strategies in our efforts to transform rare disease care. This work, led by the Innovation and Value Initiative and @Alexion, AstraZeneca Rare Disease, featuring critical caregiver perspectives from @Kelly Maynard, President of @Little Hercules Foundation, spotlights the importance of collective efforts in tackling rare disease research. By placing patients at the center of our research endeavors, we aim to pioneer innovative solutions and enhance the quality of care for those with rare conditions. Let's continue to work on bridging gaps and empowering patients on their journey towards better health outcomes. Together, we can make a tangible difference in the lives of those affected by rare diseases. #RareDiseaseResearch #PatientCentricity #InnovationAndValue
"Bridging the Gap, Empowering Patients: A Collaborative Approach to Rare Disease Research" highlights the significance of adopting a collaborative, patient-centered approach in rare disease research. Led by the Innovation and Value Initiative and Alexion Pharmaceuticals, Inc. AstraZeneca Rare Disease, the conversation underscores the importance of transforming the future of rare disease care through collective efforts and patient-centric strategies. Little Hercules Foundation Adrian Kielhorn Kaitlyn MacCarrick @Richard Chapman Watch: https://lnkd.in/e-8jHvNk
Bridging the Gap, Empowering Patients: A Collaborative Approach to Rare Disease Research
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📢 On Rare Disease Day, we support those affected by rare diseases. We're here to help biopharma partners bring innovative therapies to vulnerable patients and their families. Join us in raising awareness and making a difference! Read our article "Nine Questions about Rare and Orphan Drug Commercialization" for insights on navigating this complex field: https://ow.ly/KXiZ50QIKRX. Together, let's drive innovation and improve lives. 💙 #RareDiseaseDay
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