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https://news.gatech.edu/news/2026/03/20/atdc-startups-secure-rare-fda-breakthrough-device-status ATDC Startups Secure Rare FDA ‘Breakthrough Device’ Status | News Center It’s uncommon for any startup to receive the Food and Drug Administration’s (FDA) Breakthrough Devices designation. For the roughly 40% of applicants who... atdc startups secure rare fda status news center https://nephrodite.com/newsroom/atdc-startups-secure-rare-fda-breakthrough-device-status/ ATDC Startups Secure Rare FDA ‘Breakthrough Device’ Status - Nephrodite Mar 25, 2026 - It’s uncommon for any startup to receive the Food and Drug Administration’s (FDA) Breakthrough Devices designation. For the roughly 40% of applicants who... atdc startups secure rare fda status nephrodite https://www.gatech.edu/news/2026/03/20/atdc-startups-secure-rare-fda-breakthrough-device-status ATDC Startups Secure Rare FDA ‘Breakthrough Device’ Status It’s uncommon for any startup to receive the Food and Drug Administration’s (FDA) Breakthrough Devices designation. For the roughly 40% of applicants who... atdc startups secure rare fda status https://atdc.org/atdc-startups-secure-rare-fda-breakthrough-device-status/ ATDC Startups Secure Rare FDA ‘Breakthrough Device’ Status - ATDC Mar 26, 2026 - Milestone designation signals strong potential to reshape care for dialysis patients and those with chronic knee pain. March 20, 2026 - It’s uncommon for any... atdc startups secure rare fda status https://www.biospace.com/fda/rare-disease-leaders-call-for-regulatory-clarity-as-fda-balances-urgency-with-rigor Rare Disease Leaders Call for Regulatory Clarity as FDA Balances Urgency With Rigor - BioSpace Apr 2, 2026 - With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter... rare disease leaders call regulatory clarity fda balances https://kevinmd.com/2025/07/fda-delays-could-end-vital-treatment-for-rare-disease-patients.html FDA delays could end vital treatment for rare disease patients A patient with primary mitochondrial myopathy urges the FDA to approve Elamipretide, a life-sustaining treatment, warning that delays could halt its production... rare disease patients fda delays could end vital treatment https://medcitynews.com/2026/04/travere-therapeutics-filspari-sparsentan-fda-approval-rare-kidney-disease-proteinuria-fsgs-tvtx/ Bouncing Back From Trial Failure, Travere Wins First FDA Approval in Rare Kidney Disease - MedCity... Apr 16, 2026 - Travere Therapeutics’ Filspari is now the first FDA-approved drug for focal segmental glomerulosclerosis (FSGS), a rare kidney disease. Analysts project the... rare kidney disease bouncing back trial failure wins first fda approval https://www.statnews.com/pharmalot/2026/03/26/fda-rare-disease-pto-novo-nordisk-340b-abortion-hiv/ Pharmalittle: FDA approves rare disease drug, PTO chief grilled | STAT Mar 26, 2026 - A bipartisan quartet of senators unveiled legislation to cap the monthly cost of insulin at $35 for patients on private insurance. rare disease drug pharmalittle fda approves pto chief https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs Rare Pediatric Disease Designation and Priority Review Voucher Programs | FDA FDA awards rare pediatric disease PRVs to sponsors of rare pediatric disease products that are approved and meet certain criteria. Prior to submitting a... rare pediatric priority review programs fda disease designation https://www.biospace.com/fda/fda-ties-amgens-rare-inflammatory-disease-drug-to-eight-deaths-dozens-of-liver-injuries FDA Ties Amgen’s Rare Inflammatory Disease Drug to Eight Deaths, Dozens of Liver Injuries - BioSpace Apr 1, 2026 - The FDA in January asked Amgen to pull Tavneos from the market, citing liver toxicity issues that affected the drug’s overall risk-benefit profile. The pharma... inflammatory disease fda ties rare drug https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program Rare Disease Endpoint Advancement Pilot Program | FDA FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel efficacy endpoint development for drugs that treat rare diseases.... rare disease pilot program endpoint advancement fda https://www.biospace.com/fda/prasad-out-at-fda-lawmaker-takes-action-on-rare-disease-rejections-and-a-spate-of-obesity-data Prasad Out at FDA, Lawmaker Takes Action on Rare Disease Rejections, and a Spate of Obesity Data -... Mar 16, 2026 - Rare disease biotech stocks pop on the news that Vinay Prasad, the FDA’s chief biologics regulator, will depart the FDA at the end of April; Sen. Ron Johnson... takes action rare disease obesity data prasad fda https://nationalhealthcouncil.org/webinars/a-look-at-rare-disease-listening-sessions-with-nord-and-the-fda/?r_nkey=32bb90e8976aab5298d5da10fe66f21d&r_page=2 A Look at Rare Disease Listening Sessions with NORD and the FDA - National Health Council Mar 24, 2020 - A Look at Rare Disease Listening Sessions with NORD and the FDA This webinar featured Debbie Drell, Director of Membership, at the National Organization for... national health council rare disease listening sessions look nord https://www.npr.org/transcripts/nx-s1-5795526 A gene therapy for a rare form deafness receives FDA approval : NPR Apr 23, 2026 - The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical milestone. receives fda approval gene therapy rare form deafness npr https://www.statnews.com/2026/02/23/fda-rare-disease-new-guidelines-plausible-mechanism-pathway/ FDA unveils new pathway for rare disease treatments | STAT rare disease treatments unveils new fda pathway stat https://www.statnews.com/2026/02/26/rare-disease-treatment-approval-fda-mixed-messages/ Rare disease advocates perplexed by mixed messages at the FDA | STAT Feb 26, 2026 - FDA has rejected or reversed course on five gene therapies for rare diseases while simultaneously unveiling expedited pathways for new treatment approvals. rare disease mixed messages fda stat advocates perplexed https://www.statnews.com/2026/03/10/ron-johnson-investigating-fda-rare-disease-drug-denials/ GOP Sen. Johnson investigating FDA rare disease drug rejections | STAT Mar 10, 2026 - Sen. Ron Johnson has long been an advocate for giving people with rare diseases speedy access to experimental treatments. rare disease drug gop sen johnson investigating fda https://www.statnews.com/2026/03/10/fda-says-leucovorin-evidence-lacking-for-autism/ FDA approves leucovorin for rare disorder, not for autism | STAT Mar 10, 2026 - The FDA action represents a retreat from top officials’ earlier insistence that leucovorin could benefit fda approves rare disorder leucovorin autism stat https://www.intelligentliving.co/personalized-medicine-fda-gene-edit/ Personalized Medicine Evolution: How the FDA Plausible Mechanism Framework Impacts Ultra-Rare Gene... Apr 27, 2026 - FDA's Plausible Mechanism Framework speeds individualized gene therapy review for ultra-rare diseases, prioritizing biological proof over large clinical trials. personalized medicine ultra rare evolution fda plausible https://www.fda.gov/news-events/press-announcements/fda-launches-framework-accelerating-development-individualized-therapies-ultra-rare-diseases FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare... The U.S. Food and Drug Administration today issued draft guidance for sponsors seeking approval for targeted individualized therapies by generating substantial... fda launches accelerating development ultra rare framework individualized https://www.npr.org/2026/04/23/nx-s1-5795526/deafness-gene-therapy-regeneron A gene therapy for a rare form deafness receives FDA approval : NPR Apr 23, 2026 - The treatment, developed by Regeneron Pharmaceuticals, is for a very rare form of deafness. But it represents a medical milestone. receives fda approval gene therapy rare form deafness npr https://newsroom.ucla.edu/releases/fda-approves-gene-therapy-leukocyte-adhesion-deficiency-l-pediatric-disease FDA approves gene therapy for LAD-I, a rare immune disorder | UCLA All the children treated in a clinical trial are living with restored immune function. fda approves gene therapy rare immune lad disorder https://nationalhealthcouncil.org/webinars/a-look-at-rare-disease-listening-sessions-with-nord-and-the-fda/ A Look at Rare Disease Listening Sessions with NORD and the FDA - National Health Council Mar 24, 2020 - A Look at Rare Disease Listening Sessions with NORD and the FDA This webinar featured Debbie Drell, Director of Membership, at the National Organization for... national health council rare disease listening sessions look nord https://patientworthy.com/2026/04/05/draft-guidance-from-fda-introduces-important-changes-to-drug-development-for-rare-diseases/ Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases –... draft guidance important changes drug development rare diseases fda https://www.npr.org/2026/02/23/nx-s1-5720948/fda-rare-disease-gene-therapy FDA says a plausible mechanism can be enough for rare disease approval : NPR Feb 23, 2026 - The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical... fda says rare disease approval npr plausible mechanism https://www.biospace.com/drug-development/6-rare-diseases-that-have-new-fda-approved-treatments 6 Rare Diseases That Have New FDA-Approved Treatments - BioSpace Feb 28, 2025 - ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C,... new fda approved rare diseases 6 treatments biospace