Season's Greetings and warm wishes for the New Year from Abeona Therapeutics! We're deeply thankful for the incredible support and collaboration within the epidermolysis bullosa community this year. Click here bit.ly/46G4uky to discover more about RDEB, often referred to as the worst disease you've never heard of. Abeona's BLA submission for pz-cel has been accepted by the FDA, marking a milestone with a PDUFA target action date of May 25, 2024. Read our press release for further details bit.ly/47SH5NZ #RDEB #raredisease #EBLife #Abeonatherapeutics #FightEB #EBawareness #NewYear #HappyNewYear #SeasonsGreetings
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FDA Approval Alert! Niemann-Pick disease Type C (NPC) disease received first approved treatment in the USA on 20 Sep 2024 with approval of Miplyffa from Zevra Therapeutics. The indication is poised for a market share tussle with another competitor vying for approval, as IB1001 from Intra Bio awaits FDA decision on 24 Sep 2024!! Like and Follow Pharma Insights Buddy for more insightful content and knowledge sharing by the industry experts!! Do you want to share your insights, learnings with like minded audience? Just send your content to connect@pharmainsightsbuddy.com. Get published, reach beyond network and earn #Amazon_Vouchers if the post receives more than 50 Likes 😍 #Post_Credit: Big thanks to Anuj Kumar Rai for the analysis and sharing with PIB community.
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I’m very pleased to share this terrific news. Today’s approval by the FDA of Akebia’s oral therapy for the treatment of anemia due to Chronic Kidney Disease (#CKD) in adult patients on dialysis in the U.S. offers us a unique opportunity to provide innovative medication to patients suffering from renal disease and alleviate their burden. My warmest congratulations to our partner, Akebia Therapeutics, and all those colleagues whose hard work and dedication were instrumental in reaching this important milestone. Anemia is a condition which commonly occurs in people with CKD because their kidneys do not produce enough erythropoietin, a hormone that helps regulate production of red blood cells. Anemia due to CKD can have a profound impact on a person’s quality of life.1 Left untreated, it can lead to deterioration in health and is associated with increased morbidity and mortality.2 Today's milestone further emphasizes our commitment to patients and public health. I encourage those interested to check out the link to the press release below for further details. #collaboration #innovation #nephrology #publichealth #csl 1. Eriksson D, et al. BMC Nephrol. 2016;17:97; Finkelstein FO, et al. Clin J Am Soc Nephrol. 2009;4:33-38; Farag YM, et al. Clin Nephrol. 2011;75:524-533. 2. Portolés J, et al. BMC Nephrol. 2013;14:2. 3. NICE. Clinical Guideline: Anaemia Management in Chronic Kidney Disease: Partial Update 2015. 4. Silverberg DS, et al. Clin Lab Haematol. 2001;23:1-6. 5. Herzog CA, et al. J Card Fail. 2004;10:467-472.
We congratulate our partner Akebia Therapeutics for achieving FDA approval for their oral therapy. This represents an important moment in our shared efforts of improving the lives of dialysis patients with anemia due to chronic kidney disease in the U.S. Read more here https://bit.ly/3PGad4n #PatientFocus #CSLVifor
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On February 3, 2023, the Wiskott-Aldrich Foundation convened the Externally Led-Patient Focused Drug Development (EL-PFDD) meeting to educate the FDA and other stakeholders on how X-Linked Thrombocytopenia and Wiskott-Aldrich Syndrome (WAS) impacts our lives, and give our thoughts on current and future therapies. More than 300 individuals participated in the live webcast event, and patients and family members testimony has been captured in the 𝐕𝐨𝐢𝐜𝐞 𝐨𝐟 𝐭𝐡𝐞 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐑𝐞𝐩𝐨𝐫𝐭. This landmark report documents the severe disease burden and unmet medical need in patients' own voices. The Wiskott-Aldrich Foundation wants the FDA and all the stakeholders to listen to our patients' voice, their words, and their experiences, and recommends that these findings be considered when developing potential XLT/WAS therapeutics. The 𝐕𝐨𝐢𝐜𝐞 𝐨𝐟 𝐭𝐡𝐞 𝐏𝐚𝐭𝐢𝐞𝐧𝐭 𝐑𝐞𝐩𝐨𝐫𝐭 has been submitted to the FDA for inclusion in the framework used to evaluate future therapies for XLT and WAS. The report is here: https://lnkd.in/gCEpUB-3 The report, video of the live webcast, are posted below. Our community owes a deep debt of gratitude to the patients and family members who courageously shared their stories, took the time to attend the meeting and to answer questions about how XLT/WAS has impacted their lives and what improvements they would like to see. https://lnkd.in/gCEpUB-3 #WiskottAldrichSyndrome #wiskottwarrior #primaryimmunodeficiency #PID #primaryimmunodeficiency #wiskottaldrichsyndromeawareness #primaryimmunedeficiency #waslife #lowplateletcount #eczema #ELPFDD #PFDD #voiceofpatientreport
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#Bronchiectasis is a chronic condition affecting approx. 370,000 adults in the US, where the lung’s bronchi become permanently damaged. On #WorldBronchiectasisDay, we are raising awareness for non-cystic fibrosis bronchiectasis (NCFB), a disease with no FDA-approved drugs specifically for NCFB. Verona’s investigational drug #ensifentrine is currently in Phase 2 clinical studies for the treatment of NCFB. Learn more about this focus area: https://bit.ly/3z3gVvX
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Submit your abstract for #IgNS2024 today!
Share your research at IgNS 2024! Submit an abstract on Ig, biologics, disease management, practice innovation, clinical studies, and more by June 21, 2024. Don't miss this opportunity to present and discuss your data with the top clinicians in our field. Visit https://lnkd.in/d8sTpqXk today! #Abstracts #IgNS2024 #research #posters
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🌟 Exciting Update! 🌟 We have received FDA approval for a new Phase 3 clinical trial! Thanks to the promising outcomes from our Phase 0/2 study in collaboration with GSK, we're fast-tracking the Gliofocus Study. This global trial will compare niraparib against temozolomide in treating newly diagnosed MGMT-unmethylated #glioblastoma. 🌍 Learn more: https://loom.ly/ckeho64
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Have you read 'The impact of RARE disease on siblings experience' impact report? Supported by Alexion Pharmaceuticals, Inc. and created by RARE Revolution Magazine. The report shares the personal insights of young people living with rare disease, how it's embedded into the fabric of their family life, its impact on their education and decision-making. Read the full report at https://lnkd.in/eGTfXCyk
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🚨 Update on Zeposia Phase 3 Trials for Crohn's Disease 🚨 Bristol Myers Squibb failed to meet primary endpoint of Phase 3 induction trials of the Phase 3 YELLOWSTONE clinical trial of #Zeposia for mod-to- sev CD 📊 Strategy: ⏩No S1P modulators have yet shown significant therapeutic benefits for CD ⏩Mitsubishi Tanabe Pharma's amiselimod, undergoing Phase 2 trials, didn't demonstrate significant improvement compared to placebo ⏩The approval of Velsipity and Zeposia for UC suggests that the potential for S1P modulators may be limited to UC 📅 What's Next? ⏩The company plans to unveil full results of the YELLOWSTONE trials in the future, although no specific timeline has been provided 🔜 Stay tuned for updates! Additionally, mark your calendars for the #DDW conference happening from May 18-21, 2024, where we anticipate further insights into the YELLOWSTONE program Bristol Myers Squibb #BMS #CrohnsDisease #Zeposia #ClinicalTrials #Healthcare Source: https://lnkd.in/gCSUf3HS
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For those of you attending the American Thoracic Society Meeting (ATS 2024) in San Diego this week ..if you are pursuing clinical development of investional drugs or devices for Sepsis or its complications I would be interested in chatting or meeting in person .. I led the only Phase III clinical trial of a drug that resulted in FDA approval for sepsis (XIGRIS in 2001). I also played a key role in sepsis trials of recombinant soluble thrombomodulin, anti- TNF monoclonal Ab (Cytofab, Talactoferrin, TLR4 antagonist (E5564) , rTFPI and others #sepsis #ARDS #endotoxin #TNF, #ATS2024
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Rare diseases are more common than you think. Each year, #RareDiseaseDay raises awareness of rare diseases and recognizes more than 30 million U.S. patients who are affected. Despite the successes and progress towards fighting the thousands of known rare diseases which exist, there is more work to do. Biopharmaceutical researchers are dedicated to advancing medicine to help rare disease patients live long, healthy lives with more innovative treatment options in the pipeline. Learn more: https://bit.ly/3UTsXAQ
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