Various organizations and individuals are advocating for advancements in rare disease treatments. Moderna, known for its COVID-19 vaccine, refocuses on rare disease treatments. The FDA faces calls to streamline regulations for rare disease drug development. Collaborations and research efforts aim to improve understanding and treatment access for rare diseases.
If you receive devastating news that your child has a serious medical condition, you hope there’s treatment options for them. That's why I met with @ACHA_Heart to discuss legislative efforts to increase funding for research & education about congenital heart disease. https://t.co/QDMHb0nWnW
The Creating Hope Reauthorization Act would extend the FDA’s authority to issue Priority Review Vouchers to encourage treatment for rare pediatric diseases. I co-lead this bill to ensure rare disease patients have access to future medications. https://t.co/1cZnPaMaNN
In today’s Energy and Commerce Committee Hearing @energycommerce, we discussed legislative proposals to support patients with rare diseases and the need to expand access to care for patients with rare diseases including our most vulnerable patients – kids. https://t.co/TFe48KUNdK
Time is of the essence for children and adults with rare diseases who need access to specialized treatments and life-saving therapies. Today in @EnergyCommerce, I urged my colleagues to support measures that reduce barriers to care for patients with medically complex needs. https://t.co/4F8u70fwl4
Our incredible Patient Senate at @patientsrising , made up of patients, advocates & caregivers, volunteer their time to champion patient-centered policy. Thank you @RepMMM for submitting their statement to amplify solutions for Rare Patients. #RareDC2024 https://t.co/uETK2vXviH
As a hospital that puts children with rare and complex conditions at the heart of what we do, it was fantastic to see the Government launch the 2024 Rare Diseases Action Plan and host @Andrew4Pendle at our @NIHRresearch BRC and CRF #RareDiseaseDay https://t.co/D8fEU4Hpy5
A Comment article in @MethodsPrimers discusses how optimal study design and partnerships between academia and industry are needed for successful development, delivery and clinical approval for therapies to treat rare diseases . #RareDiseaseDay https://t.co/xNaAISC9t1 https://t.co/ORpnfsRUJT
The FDA is committed to advancing treatment options for patients with rare diseases & engaging stakeholders to help address the challenges associated with rare disease research and, when applicable, incorporate the patient perspective in medical product development & regulation. https://t.co/82sw8ZDznb
Our incredible Patient Senate at @patientsrising , made up of patients, advocates & caregivers, volunteer their time to champion patient-centered policy. Thank you @MMM for submitting their statement to amplify solutions for Rare Patients. #RareDC2024 https://t.co/0Bv4n16eMS
On this special #RareDiseaseDay blog, we're inspired by the work of our customers, including @radboudumc, who are using #PacBio HiFi sequencing to help advance potential diagnostic research capabilities for challenging #RareDisease cases. https://t.co/koWl8dWdt6
Grateful to do our small part collaborating with clients who work to improve the quality of life for patients with Duchenne, myotonic dystrophy, Angelman’s Syndrome, and rare brain tumors. Over 300 million people globally live with a rare disease. 🔴🧠 #RareDiseaseDay https://t.co/OnnrgoyVc1
In addition to the Nanopore Community, we’ve seen a year of game-changing collaborations, including partnerships with @GenomicsEngland, @GeneyxGenomex, @FabricGenomics, @seqonegenomics, @saphetor & more, to accelerate rare disease research: https://t.co/kMLXPkFYDX #RareDiseaseDay https://t.co/Ee5l7j1dIR
Learn how the FDA is helping to deliver meaningful advances to rare disease patients in the latest #FDAVoices article: https://t.co/kHpUPx8lvw https://t.co/hJWZBy8ui6
Over 300 million people are affected by a Rare Disease worldwide. Children make up nearly half of those living with rare diseases, yet children's treatment options are extremely limited. Proud to co-lead the Creating Hope Reauthorization Act, which will help with the… https://t.co/NjQmUs4jnp
Revealing more biology to transform human health. From antithrombin deficiency to neuropathies, researchers across the globe are identifying genetic variations — putting the future of healthcare in the hands of scientists. Learn more: https://t.co/lplulOb5E2 #RareDiseaseDay https://t.co/f9Ihk3USQ9
A Review in @NatureRevGenet explores emerging approaches for the identification of disease-causal genetic variants as well as omic technologies that show great potential for variant interpretation. #RareDiseaseDay https://t.co/meiuqEr4zR https://t.co/giO8P0F8RO
We're proud to support investments to build, shape and strengthen #RareDisease research in the UK, including a landscape review and establishing a £14m UK Rare Disease Research Platform with @NIHRresearch, so we can better support those affected. More: https://t.co/0OnFUdMhQv https://t.co/lbepbck0vw
Rare-disease researchers pioneer a unique approach to clinical trials. A recent news feature from @TechTyper, part of our series Rethinking Evidence in Medicine. #RareDiseaseDay https://t.co/Z6EsojBgMM
The Zayed Centre for Research into Rare Diseases in Children continues to bring together pioneering research and world-leading clinical care. The centre tackles some of the most challenging children’s diseases - it's great to witness the work they do on a daily basis. https://t.co/8cT8Rlycfm
Rapid access to genomic information can change lives. Emma Baple discussed the potential of using nanopore tech to rapidly detect genetic variants & to reduce time for genetic results in critical care settings in the future. Learn more: https://t.co/56Qw2A3gC9 #RareDiseaseDay https://t.co/qN9qxD5Mny
Today is Rare Disease Day. Read about immediate changes the @US_FDA could make to give hope & access to treatment to millions suffering from rare diseases. https://t.co/N0V7XgAZZt
Today, on Rare Disease Day, I stand proud as part of Advanz Pharma. Our commitment extends beyond merely developing treatments; it's about profoundly understanding the impact of our work on real lives. Here's to making every day a step towards unlocking new possibilities in the… https://t.co/SAdPFLTbRt
Today, on Rare Disease Day, I stand proud as part of Advanz Pharma. Our commitment extends beyond merely developing treatments; it's about profoundly understanding the impact of our work on real lives. I am reminded today of the crucial importance of what we do. Here's to making… https://t.co/m679SxgxD4
Despite progress in treating rare diseases, including widening the insurance net and streamlining foreign drug approvals, patients are still grappling with high costs and the complexities of importing essential medications. #RareDiseaseDay https://t.co/zJ5D97rQg0
Despite progress in treating rare diseases, including widening the insurance net and streamlining foreign drug approvals, patients are still grappling with high costs and the complexities of importing essential medications. #RareDiseaseDay #RareDiseaseDay2024…
On #RareDiseaseDay we’re celebrating the #Nanopore Community dedicating their research improving our understanding of #RareDisease. Discover how they’re generating ultra-rich nanopore reads to generate answers with real impact. Learn more: https://t.co/4ZGTcEGL1A #wymm
.@danrdanny & @AhmadTayoun's research is revealing hidden variants unresolved by traditional methods, & the potential of nanopore sequencing to address the many challenges in identifying the molecular basis of rare diseases. Learn more: https://t.co/fr9Fo50Ps1 #RareDiseaseDay https://t.co/DGXw3Fm1Bi
From our latest Editorial: ‘A collective approach involving integration of rare diseases into the health-care system, inclusion of mental health considerations in rare disease services, and increased awareness efforts will be essential’ #RareDiseaseDay https://t.co/hR4mPcgGid https://t.co/JQL2m2mgLG
On #RareDiseaseDay we’re celebrating the #Nanopore Community dedicating their research improving our understanding of #RareDisease. Discover how they’re generating ultra-rich-rich nanopore reads to generate answers with real impact. Learn more: https://t.co/pot1NIBnma #wymm https://t.co/X05EBfbXOU
Reagan-Udall Foundation meeting could be turning point for rare disease drug development. Patients, companies, academics urge @US_FDA to accept biomarkers for ultra-rare MPS disorders. https://t.co/9Mb4nJgJLN
As co-chair of the Rare Disease Congressional Caucus, I always appreciate the opportunity to meet families and individuals impacted by rare diseases. I met with Mississippi advocates with the EveryLife Foundation to learn how Congress can better support them. https://t.co/fEAQfpwvl0
As with many rare diseases, critical treatments are often lacking. The good news is that a number of companies are working in this arena with candidates in clinical trials. Learn more: https://t.co/uKgtyWydYF https://t.co/JjWqrn39ck
Rare Disease Day: Moderna Gets Back to Its Roots After being diverted by the SARS-CoV-2 pandemic to develop and manufacture COVID-19 vaccines, the mRNA therapeutics company has refocused on advancing #raredisease treatments through the clinic. Learn more: https://t.co/j42OaokwXG https://t.co/Cm26DDs1qW
What if we could use #AI to help people with rare diseases? Our new MATRIX project will use this technology to understand how already approved drugs could be effective against other diseases, and we're working with @EveryCure to support this research. https://t.co/CmkgqpEpOP https://t.co/8xU2YDo6J7
📣HAPPENING NOW: @WHOSTP hosts White House Rare Disease Forum. @ARPA_HDirector and leadership from @WHOSTP, @NIH, @ncats_nih_gov, @HHSgov, and more will discuss developments and opportunities supporting patients and families facing a rare disease. https://t.co/PsEDizVDHN
From @WSJopinion: The pace of science is accelerating, but people struggling with rare diseases won’t see that progress if the regulatory state can’t keep up, writes @Judy__Stecker https://t.co/9ioO1tyxHy
Mayo Clinic Center for Individualized Medicine researchers are harnessing emerging technologies, individualized therapies and AI to drive meaningful progress for the 300 million people worldwide who have a rare disease. Learn more: https://t.co/v1XBwUGE7s
Today, we are proud to announce that we have joined the RARE Corporate Alliance @GlobalGenes along with many other #lifesciences companies to drive progress in #raredisease research and improve access to treatment. https://t.co/utyX60SEtt
Yesterday, rare and progressive disease advocates, patients, and families came together to support the Promising Pathway Act. Patients need access to treatment now. They don’t have time to wait. Contact your Senators and Representatives to support the #PromisingPathwayAct! https://t.co/GdOZAg4kfD
Rare Disease Day: Moderna Gets Back to Its Roots Even before its RNA vaccine for COVID-19 made Moderna famous, the company's founders had a vision for treating rare diseases. Learn more: https://t.co/j42OaokwXG https://t.co/qidOuoznDh
The FDA Could Help Save My Son From a Rare Disease by @Judy__Stecker. As this piece details, Biden socialist price controls and FDA corrupt over-regulation suppress drug innovations that could help countless Americans. https://t.co/Y2Ro00OiiM via @WSJopinion