RS BioTherapeutics, Inc.的动态

Quick tricks to improve patient care!

?? A Major Win for the Cystic Fibrosis Community! A few months ago Vertex Pharmaceuticals and the NHS reached a pricing agreement, expanding access to transformative CF drugs like Kaftrio. This milestone is deeply personal to me; my cousin Rebecca, who’s battled CF for 30 years, is among those benefiting from Kaftrio. It has truly changed her life. As I work with life sciences leaders and advocate for CF advancements, this progress is particularly inspiring. Here’s to continued breakthroughs in CF care! ?? #CysticFibrosis #LifeSciences #HealthcareInnovation #VertexPharmaceuticals #Biotechnology

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 [email protected]. 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.

My latest from Drug Topics:

Breaking Down Soliris' Success: A Playbook for Orphan Drug Launches ?? How did Soliris, an orphan drug, defy payer resistance and growing biosimilar competition to achieve blockbuster status? Our latest case study explores the strategic moves that propelled Soliris to the forefront of the rare disease market. Explore these critical insights to shape your own launch strategies, watch the video here ?? https://lnkd.in/dT7Mcucr #OrphanDrug #Soliris #RareDisease #LaunchStrategies #Biopharma #PatientSupport #PharmaceuticalInnovation #BestPracticesLLC #MarketAccess #HealthcareMarketing #Alexion

Today?we?participated in a meeting?hosted?by?the?Foundation for Sarcoidosis Research that was a unique opportunity to hear firsthand the impact of?this?disease on?patients’?lives. It was an?important venue for informing the?FDA on the significant unmet need in sarcoidosis and the need for flexibility in drug product approval approaches. ? The formation and persistence of granulomas of unknown etiology in one or more organs is the hallmark of sarcoidosis, leading to chronic inflammation, debilitating symptoms, organ disfunction, and sometimes organ failure.?Sarcoidosis affects twice as many black people as white people,?and twice as many women as men.?For some, sudden cardiac arrest is the first symptom that leads to a diagnosis.??People living?with sarcoidosis report experiencing pain, breathlessness, fatigue, brain fog, depression, anxiety and other symptoms that adversely affect their ability to function, stay gainfully employed,?and?perform normal daily activities.?There are currently no approved medicines demonstrated to be effective and safe for sarcoidosis, and the available medicines used off-label often come with toxic side effects. ? These patient?stories deeply motivate teams like ours at Kinevant to power through the challenges of conducting clinical research in rare diseases?like sarcoidosis to provide a better treatment option.?Namilumab is a once-monthly injection that could, if successfully developed and approved, be administered by sarcoidosis patients at home.?We’ve officially completed the last visit for the last patient enrolled in our?Phase 2?RESOLVE-Lung study for?pulmonary sarcoidosis.?We’re hopeful the results?of this study?expected end of the year will enable us to continue development and eventually provide these patients with a new therapy?that gives them back a normal life.

How often do you see a news report of an amazing medical breakthrough in a life-threatening disease or illness?? ? In reality, it takes precious time for a promising medicine to reach the patient who desperately needs it.? ? To explore and determine the principles that might be considered when establishing processes to provide faster access to life-saving treatments, Bellberry invited representatives of regulators, reimbursement agencies, pharmaceutical companies and, most importantly, patient groups from around the globe to Adelaide, Australia for an International Scientific Congress.? ? Tune into this edition of From Lab To Life to hear from some of the experts who attended this Congress as they grapple with this complex issue - Lloyd Sansom, Edward Cliff, Brian O'Rourke, Ann Single, Ian Kerridge, Joshua M. Sharfstein, Chris Henshall, Dan Ollendorf, Michelle Mujoomdar, PhD, Yasuhiro Fujiwara, Bettina Ryll, Meindert Boysen PharmD MSc, Christoph Glaetzer, Dipl.Kfm. ?? ? Bellberry acknowledges meeting support provided by Amgen, AstraZeneca, Johnson & Johnson and MSD. The Final Report available through this link – https://lnkd.in/gr5dEEem also provides more detail on this issue and recommendations from the Congress. https://lnkd.in/gJek_YR4 #FromLabToLife #Bellberry #ResearchEthics

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

Today marks a significant milestone in our mission to create an accessible and diverse clinical trial ecosystem. EQBMED proudly welcomes Amgen and Merck as our inaugural sponsors. Amgen and Merck will co-design and pilot strategies—such as enhancing infrastructure, increasing trial capacity, and providing support to overcome barriers like travel and transportation—to foster closer engagement with local EQBMED trial sites and communities. This collaboration is key to improving medical research by bringing local trial sites, communities, and sponsors together as partners. https://lnkd.in/er4uYQEN

Agreed, the patient-centric approach is so important when it comes to treatments.