The #FDA has sketched out its new #regulatory pathway to market for individualised therapies that treat #ultrararediseases, based on a 'plausible mechanism' premise.

pharmaphorum.com/news/fda-map...

"From a patient perspective, @realised-ihi.bsky.social can be a gamechanger," said patient advocate Ines Alves. Check out the video below to learn how this new project is improving #ClinicalTrials for #UltraRareDiseases. 👇
#IHITransformingHealth #EUResearch #HorizonEU

UCB reports success in phase 3 study of fenfluramine for CDD - PharmaTimes Positive trial results offer hope for those with ultra-rare epileptic disorder

#UCB #fenfluramine #CDD #ultrarareepilepticdisorder #ultrararediseases #CDKL5deficiencydisorder #developmentalandepilepticencephalopathy #DEE #earlyonsetepilepsy #neurodevelopmentaldelays #developmentalandepilepticencephalopathies #CDKL5gene
pharmatimes.com/news/ucb-rep...

New drug offers potential cure for ultra rare inherited condition A teenage patient who helped discover a rare condition is the first to benefit from a new treatment.

#rarediseases #ultrararediseases #immunology #Joenja #leniolisib #PharmingTechnologies #raredisease #ActivatedPI3kinaseDeltaSyndrome #APDS #UniversityofCambridge #Addenbrookes #AnitaChandra #disregulatedimmunesystem #APDSpatients #SpecialisedCommissioning
www.bbc.co.uk/news/article...

Changes approved to the routing criteria for highly specialised technologies

#NICEnews #NICE #highlyspecialisedtechnologies #NICEevaluations #NICEdecisions #HSTprogramme #ultrararediseases #routing #NICEroutingcriteria #RareDiseasesActionPlan #rarediseases #costeffectivenessthreshold #NHS #NHSresources #NICErecommendation
www.nice.org.uk/news/article...

What are the biggest challenges in ultra-rare diseases?
👨‍🔬 Ralf Dieter, Scientific Coordinator of #RealiseD, shares how the initiative is tackling these challenges.
🚀 Join us on this journey to transform the lives of millions of people living with rare and #UltraRareDiseases!

Ultra-Rare-Disease Groups Get a Boost from Chan Zuckerberg Initiative Just-announced Rare As One grants from the Chan Zuckerberg Initiative will have a big impact at nonprofits like the mitochondrial disorder patient group CureARS.

🧬 The Chan Zuckerberg Initiative is boosting support for ultra-rare disease patient groups, accelerating research and access to care.
www.insidephilanthropy.com/home/ultra-r...

#Philanthropy #UltraRareDiseases #ChanZuckerbergInitiative #SocialImpact #MedicalResearch