National Psoriasis Foundation’s Post

Discover the challenges and opportunities in the rare disease market with our latest white paper. Gain insights on launching orphan medicines, overcoming hurdles and achieving #launchsuccess. Download now to learn from successful #RareDisease launches. https://bit.ly/3yaeo2u #LaunchExcellence

Getting a lot of positive reaction with our new white paper on Rare disease Launch Excellence..see below.

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.

Poolbeg Pharma plc CEO Jeremy Skillington joined Steve Darling from Proactive to announce the company’s unaudited interim results for the first half of 2024, highlighting strong progress with its lead development candidate, POLB 001. As of 30 June 2024, the company reported a cash balance of £10.1 million, showcasing its prudent financial management and ability to support ongoing development initiatives. Skillington emphasized that POLB 001 is on track, with robust data demonstrating its efficacy in reducing cancer immunotherapy-induced cytokine release syndrome (CRS) in an in vivo animal model. This strengthens the company's patent portfolio, with the U.S. Patent Office recently granting Poolbeg a new patent for its Immunomodulator II, covering a class of drugs, including POLB 001, aimed at treating or preventing hypercytokinemia (cytokine storm) across a range of diseases. This patent approval enhances Poolbeg’s ability... Watch at #Proactive #ProactiveInvestors http://ow.ly/ExK8105J2J7

Gain insights on launching orphan medicines, overcoming hurdles and achieving #launchsuccess. Download now to learn from successful #RareDisease launches. https://bit.ly/3yaeo2u

Cdkl5 + long term potentiation = 7 publications. BRAINCURES Discovery Engine (BDE) -Targets and -Compounds prioritizes 292 (41%) and 81 (11%) Cdkl5 publications, respectively.

Vault America sponsors the 20th Annual National Scleroderma Foundation New England Golf Classic to help cure Scleroderma. Please share this post to help to bring more awareness to Scleroderma. Scleroderma occurs three to four times more often in women than in men, and it also affects children. The disease, which literally means “hard skin” can result in thickening and tightening of the skin, as well as causing serious damage to internal organs. Scleroderma affects less than 500,000 people in the United States and is therefore considered to be an orphan disease so does not receive federal funding. #knowscleroderma #curescleroderma #scleroderma #cure4scleroderma #wescoefoundation #pfwarriors #fibrosis #pulmonaryhypertension #pulmonaryfibrosis #ChannelCon Scleroderma Foundation New England (SFNE) Pulmonary Fibrosis Foundation Arthritis Foundation The Myositis Association Wescoe Foundation for Pulmonary Fibrosis The Janssen Pharmaceutical Companies of Johnson & Johnson Horizon Boehringer Ingelheim CompTIA SecureEDEN, Inc. Anne Sweeney Jane Ladas Mirian Moultrie Mary Wheatley, IOM, CAE David Carroll Eric Bull Jeff Solomon Zak Karsan

Embracing Strength and Resilience on World Lupus Day! Today, on World Lupus Day, Alicon Pharmaceutical stands in solidarity with the global community affected by lupus, honoring the strength and resilience of individuals navigating this complex autoimmune disease. At Alicon, we're dedicated to advancing research and providing support for those living with lupus. Through innovative treatments and compassionate care, we strive to improve outcomes and enhance quality of life for patients worldwide. This World Lupus Day, let's come together to raise awareness, promote understanding, and advocate for better care and support. Together, we can empower individuals to live life to the fullest despite the challenges of lupus. Join us in our commitment to fostering hope, resilience, and empowerment in the lupus community. Together, let's shine a light on strength and inspire a future where every individual impacted by lupus can thrive. #worldlupusday #aliconpharmaceuticals #lupusawareness

🔬 Rare Disease Day 2024: Pioneering Hope for LSDs 🔬 On this Rare Disease Day, we stand united with the global community to shine a spotlight on lysosomal storage disorders (LSDs) and the ongoing journey towards groundbreaking treatments. We are privileged to be at the forefront of research and development aimed at conquering these complex conditions. LSDs, though individually rare, collectively impact the lives of millions worldwide, presenting unique challenges to patients and their families. Our mission is not just about developing treatments; it’s about nurturing hope, fostering innovation, and driving progress that transforms lives. This year, we’ve made significant strides in our research into LSDs, thanks to our dedicated team of scientists, patients’ invaluable contributions, and our collaborative efforts with healthcare providers. Our commitment is unwavering: 1. Innovative Research: We’re pioneering advanced therapies that promise to redefine what’s possible in the treatment of LSDs. 2. Patient-Centric Approaches: Our developments are guided by the real needs of patients, ensuring that our treatments are not only effective but also accessible. 3. Global Collaboration: We believe in the power of unity, working alongside global partners to share knowledge, resources, and hope. As we mark Rare Disease Day, let’s celebrate the progress we’ve made and the journey ahead. The path is long, but together, we are paving the way towards a brighter future for those affected by LSDs. To everyone battling a rare disease, we see you, we hear you, and we’re with you every step of the way. Here’s to breaking new ground, together. 🌟 #RareDiseaseDay #LSDs #InnovationInHealthcare #TogetherWeCan

🗓️ Join us tomorrow! ERGOMED webinar on "Choosing the Right CRO for Your Rare Disease Program" with Juliet (Jules) Moritz and Benjamen Varsano is fast approaching. Gain practical knowledge, learn from real-life case studies, and get your questions answered by industry leaders. Register for our webinar tomorrow here: https://lnkd.in/dQTMgNth #RareDiseaseResearch #ClinicalTrials #Webinar #CRO #ClinicalResearch