The IgA Nephropathy Foundation’s Post

Tomorrow, February 29, it’s #RareDiseaseDay. In 1983, #OrphanDrugAct was passed and brought more resources toward the research, development, and distribution of #RareDisease therapeutics. 41 years later, we celebrate the continued progress and hope within the rare disease community! If you want to support anyone with a #RareDisease, you can learn more here: bit.ly/RDD2024 #RareDiseaseDay, #LightUpForRare, #ShareYourColors, #ShowYourStripes

🚦 Green Light for Zevra 🚦 Zevra Therapeutics has secured FDA approval for Miplyffa, targeting Niemann-Pick type C disease, in collaboration with Johnson & Johnson's miglustat. This marks the first FDA-approved treatment for NPC, focusing on neurological symptoms in patients 2+ years. After a previous setback, Zevra Therapeutics is set to launch Miplyffa within 8-12 weeks, accompanied by the new AmplifyAssist support program. The approval followed promising trial results and an FDA advisory committee's favorable vote. This is a significant leap for NPC patients, with Miplyffa addressing a crucial unmet need. #RareDisease #FDAApproval #ZevraTherapeutics

FDA’s approval of MIPLYFFA™ (arimoclomol) on Friday, September 20, was a watershed moment for the Niemann-Pick disease type C (NPC) community and for Zevra Therapeutics. MIPLYFFA™ is the first FDA-approved therapy for use in patients 2 years of age and older with this ultra-rare, relentlessly progressive, degenerative, and potentially fatal disease.  XOMA Royalty is entitled to receive a mid-single digit royalty on MIPLYFFA™ sales and up to $52.6 million in milestone payments. Our press release can be found here https://bit.ly/3XUjYk7. More detailed information on MIPLYFFA™ can be found in Zevra’s press release which is available here https://bit.ly/3XS9d1t. #NPC #NiemannPick #MIPLYFFA #arimoclomol #Royalties #Royalty #Milestones #NewDrugApproval #RareDisease 

Don't miss our keynote presentation by Dr. Daniel O'Connor. He will discuss new perspectives on how the regulatory system can be used to advocate for patients and individualized therapeutics in the rare disease ecosystem. Register today! https://lnkd.in/gzaxRX8q #N1CAnnualMeeting #IndividualizedMedicine #GlobalHealthPerspective #RareDiseases #PersonalizedMedicine #RegulatorySystem

🌍 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

Can treprostinil inhalation solution (nebulized Tyvaso) make significant patient outcome change in IPF patients? When I learned about the United Therapeutic's TETON trial program, I was amazed by the company audacity. IPF and ILDs have become the treatment graveyard and we have seen multiple failures in the past, including Galapagos' ISABELA replicate studies. As we await the outcomes of the TETON studies, the possibility of a breakthrough in the treatment of IPF and ILDs remains a fervent wish, one that could herald a new era in the management of these conditions. United Therapeutics Corporation Announces Full Enrollment of the TETON 2 Study of Inhaled Treprostinil for the Treatment of Idiopathic Pulmonary Fibrosis – United Therapeutics Corporation

More than 95% of all #rarediseases lack any FDA-approved treatment options, and bringing a drug to market for a #raredisease is an inherently complicated and risky endeavor. That is where pilot programs like the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program come in, promising to potentially shave off years of the #drugdevelopment pipeline. #FDA announced the first pilot program participants earlier this week, offering much-needed hope to the 7 associated rare disease patient communities - and ultimately to everyone in the rare space who will be able to benefit from the shared learnings. Read the full article here: https://lnkd.in/eeJYSsMA

🌟 JUST RELEASED: BCOP 3rd Edition! 🌟 Showcase your dedication to superior oncology care with your BCOP Certification, and strengthen your influence in healthcare. Our newest edition BCOP Review is tailored to ensure your success on the notoriously difficult BCOP Exam, while ALSO advancing your skills as an oncology specialist, empowering you to make a lasting impact on patient care. Take advantage of *fully updated topic reviews, *expanded disease state reviews, and *405 brand new oncology Q-bank questions to boost your confidence and hone your preparation. Together, we will impact healthcare for good! . . Follow: 👉 @highyieldmedreviews 👊 Like | Comment | Share 👊 👇HASHTAGS👇 #BCOP #myhighyield #clinicalpharmacy #clinicalpharmacist #clinicalpharmacology #pharma #bpspharma #BCOPCertification #OncologyExcellence #HealthcareHeroes

On July 3rd, we’ll join CureDuchenne for a webinar about the efficacy and safety data from the DELIVER trial of DYNE-251, an investigational therapeutic for people with #DMD amenable to exon 51 skipping: https://bit.ly/3zvfpTf #clinicaltrial #drugdevelopment

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