The Ehlers-Danlos Society’s Post

We're pleased to introduce our sponsors Zevra Therapeutics who are researching vascular Ehlers-Danlos syndrome (vEDS)! "Zevra Therapeutics is committed to developing treatments for rare diseases. Our phase 3 clinical research study tests celiprolol, an investigational medicine that is being evaluated to determine its efficacy in treating vascular Ehlers-Danlos syndrome (vEDS). Learn more about the Decentralized Study Evaluating Celiprolol on VEDS Related Events (DiSCOVER) clinical trial and enroll here." https://lnkd.in/efSZrnZB #Sponsored #SponsoredPost #Ad #ClinicalResearch #RareDisease #VEDS #VascularEhlersDanlosSyndrome #VascularEDS #EDS #EhlersDanlosSyndrome

🌍 This #RareDiseaseDay, we're shining a light on the power of innovation. Sutura Therapeutics' drug conjugates platform is breaking barriers in rare disease treatment, offering new hope where it's needed most. #SuturaInnovation #HopeInEveryMolecule #SuturaTech #InCellTherapy #BiotechInnovation

Understanding the FDA’s "Not Same Active Moiety" principle is crucial for navigating the regulatory pathways for Rare Pediatric Disease Designation (RPDD) and Priority Review Vouchers (PRV). Our latest article explores how this principle ensures PRVs reward only groundbreaking therapies and maintain their role in driving innovation. Using Argenica Therapeutics as a case study, we highlight how active moiety scrutiny impacts eligibility at both the RPDD and PRV stages, providing practical insights for drug sponsors. Read the full article to learn more about this pivotal regulatory framework. #BiotechInnovation #RegulatoryAffairs #RareDiseases #PediatricHealth #PharmaStrategy #FDAApproval #KYBORA #ThePRVFundProject

BioLineRx Doses First Patient with Motixafortide in P-II Study for Treating Pancreatic Ductal Adenocarcinoma (PDAC) #biolinerx #motixafortide #pancreaticductaladenocarcinoma #clinicaltrial #phase2 #treatment #pdac #safety #efficacy #firstpatientdosing

Novel and Emerging Therapies in Psoriatic Arthritis Recent advancements in targeted therapies, including selective inhibitors of key inflammatory pathways, offer promising outcomes for patients with active psoriatic arthritis (PsA). At ACR 2024, there have been further developments in novel therapies in PsA. Three innovative treatments: Zasocitinib, Vunakizumab, and Sonelokimab, as detailed in recent phase 2 trials, were presented at #ACR24.

The Alternative pathway amplification loop is central to the essential amplification of the complement response. It provides promising targets for emerging complement therapeutics. These targets have the potential to improve disease outcomes while maintaining the complement system’s protective capabilities. Learn more how researchers are exploring drugs that target the alternative pathway to treat a wide range of diseases. https://hubs.ly/Q02CzL200 #ComplementingLifeScience #AnswersinLifeScience #Complementbiology #ComplementResearch #AlternativePathway #ComplementSystem #ImmuneResponse #ImmuneSystem #Drugdevelopment #Therapeutics #Properdin

This article, published in the latest European Biopharmaceutical Review issue, provides great insights from Ivo Timmermans and Dan Gelvan on maximising the opportunities and navigating the challenges of developing a first-in-class therapy. #PlecoTherapeutics #Firstinclass #Healthcareinnovation #EBRMagazine

We are excited to share that The Lancet published 12-month safety and efficacy data from our Phase I/II clinical trial evaluating ATSN-101 the first, investigational #GeneTherapy therapy to treat the #InheritedRetinalDisease #LeberCongenitalAmaurosis caused by biallelic mutations in GUCY2D (LCA1).   ATSN-101 demonstrated durable, clinically significant improvements in vision and was well-tolerated 12 months post-treatment. We are encouraged by these promising results, which support further evaluation in a Phase III trial. ATSN-101 has the potential to become the first-ever #GeneTherapy for LCA1, marking a significant advancement in reversing blindness that begins in childhood.   Read the full article published online in The Lancet. https://lnkd.in/eCsf8Khq   Image description: Text reads: Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase I/ll Clinical Trial of ATSN-101 in LCA1 Published in The Lancet. The Atsena Therapeutics logo is in the bottom right corner over a white bar stretching across the bottom.

Don’t miss out on our case study on the blockbuster journey of Tepezza, offering essential launch and market entry insights for the biopharma industry. Uncover how Horizon Therapeutics revolutionized thyroid eye disease treatment, setting a new standard in ophthalmic care. Watch here: https://lnkd.in/dcEfA4tw #Tepezza #RareDiseases #HorizonTherapeutics #OphthalmicBlockbuster #ThyroidEyeDisease #TepezzaCaseStudy #Pharma #HealthcareInnovation #Launch #MarketEntry #Phama #Opthalmology #BestPractices #BestPracticesLLC

The Alternative pathway amplification loop is central to the essential amplification of the complement response. It provides promising targets for emerging complement therapeutics. These targets have the potential to improve disease outcomes while maintaining the complement system’s protective capabilities. Learn more how researchers are exploring drugs that target the alternative pathway to treat a wide range of diseases: https://hubs.ly/Q02KypWz0 #ComplementingLifeScience #AnswersinLifeScience #Complementbiology #ComplementResearch #AlternativePathway #ComplementSystem #ImmuneResponse #ImmuneSystem #Drugdevelopment #Therapeutics #Properdin