karibuHow Rare Disease Patients Are Rewriting The Rules Of Medicine
PeerTube
PNH & aHUS Market Growth Report 2026–2035 📊
The Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS) market is projected to grow from $5.97B in 2025 to $6.41B in 2026, driven by rising rare disease diagnosis, advanced testing, and next-generation complement inhibitors.
Get the free sample report:
https://www.thebusinessresearchcompany.com/sample.aspx?id=24357&type=smp
Charité Berlin💡 A genuine surprise: The active ingredient in #Viagra, sildenafil, helps treat a rare genetic disorder! This unexpected finding has now been uncovered by researchers at #CharitéBerlin. The drug showed positive effects on the course of the disease in six patients with #Leigh syndrome. More on the promising results:
EU PublicationsAround 36 million patients in the EU suffer from #RareDiseases. This can have a tremendous influence on their quality of life. What does the EU do to understand and address the challenges?
See the recent initiatives and actions taken in this field https://link.europa.eu/BnKbCq
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https://nitter.net/EUPublications/status/2027715569043312868#m
See the recent initiatives and actions taken in this field https://link.europa.eu/BnKbCq
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https://nitter.net/EUPublications/status/2027715569043312868#m
Tackling rare diseases - Publications Office of the EU
There are around 36 million patients suffering from rare diseases in the EU. This in-depth analysis gives an overview of European actions for rare diseases to date and identifies opportunities and gaps for further EU level actions to improve diagnosis, care, and treatment for rare diseases. This document was provided by the Policy Department for Economic, Scientific and Quality of Life Policies at the request of the Committee on Public Health (SANT).
Politico.eu (Unofficial RSS)Q&A: Families shouldn’t have to coordinate Sweden’s rare disease care https://www.politico.eu/sponsored-content/families-shouldnt-have-to-coordinate-swedens-rare-disease-care/?utm_source=RSS_Feed&utm_medium=RSS&utm_campaign=RSS_Syndication #Advancedtherapies #Regions/Cohesion #Accountability #Communications #Healthsystems #Rarediseases #Transparency #Governance #Healthcare #Regulation #HealthCare #Hospitals #Medicines #Stability #Advocacy #Patients #Research #Services #Borders #Bridges #digital
A-Dub 🥝what it's like to have erythropoietic protoporphyria:
Rare sun disorder kept Saskatoon man indoors for decades until a clinical trial changed everything | CBC News
Kris Kinar lived 30 years with EPP, spending most of his time indoors. After a tough diagnosis in his mid-30s, he thought he would live with it for the rest of his life. In early 2025, he began a clinical trial after meeting a doctor who understood the science.
Zeimyth ΘΔAttended a community awareness event this afternoon for Rare Diseases. Conventional wisdom says that, if you hear hoofbeats, it's probably a horse. Applied to so-called "Rare Diseases", or diseases that only affect less than 1 in 2000 people, this approach results in signs and symptoms being improperly diagnosed or never diagnosed at all. Medicine is poorly equipped to help people with most rare diseases, resulting in difficulty, pain, and premature death for many people with these misunderstood conditions. In other words, these hoofbeats weren't being made by horses, they were being made by zebras.
This was my first experience really encountering this concept. At first I was skeptical about putting "Rare Disease" as an umbrella over so many conditions, but I have a better appreciation for it after today. It's very much a "strength in numbers" type of situation. None of these diseases are enough to create much noise by themselves, but together they can have a shared voice that can actually be heard.
It was also while at this event that I realized that my third child, who was stillborn and diagnosed posthumously with trisomy 13, was one such example of someone with a Rare Disease. That helped things hit a bit closer to home as well. Despite these diseases being so rare, there are also many types, with as many as 10% of people having one. That's sobering to think about.
BioResireGene therapy revolutionizes medicine by correcting defective genes and treating inherited diseases, cancers, and rare disorders with advanced biotechnology innovations.
#GeneTherapy #Biotechnology #GeneticEngineering #MedicalInnovation #PrecisionMedicine #CancerResearch #RareDiseases #BiotechStudents #LifeSciences #BioResire
#GeneTherapy #Biotechnology #GeneticEngineering #MedicalInnovation #PrecisionMedicine #CancerResearch #RareDiseases #BiotechStudents #LifeSciences #BioResire
News BeepFDA unveils new pathway for rare disease treatments
The Trump administration on Monday released detailed guidance for approving the first bespoke medicines crafted to treat patients’ individual…
#NewsBeep #News #Topstories #biotech #CRISPR #FDA #Genetherapy #gene-editing #Headlines #rarediseases #STAT+ #TopStories
https://www.newsbeep.com/406570/
@Co_BiologistsFebruary is Rare Disease Month, with Saturday 28 Feb being Rare Disease Day. To highlight this, Disease Models & Mechanisms is promoting its subject collection on rare disease research.
All articles are Open Access, so free to read and share for everyone: https://journals.biologists.com/dmm/collection/39/Rare-Disease-Translational-Research
