Rare Diseases

Aytu BioPharma adds late-stage pediatric onset Rare Disease asset to development pipeline from rumpus Therapeutics BioSpace13:29FDA Pharma News Biotechnology
In the last half-hour
Aytu BioPharma Adds Late-Stage Pediatric Onset Rare Disease Asset to Development Pipeline from Rumpus Therapeutics BioSpace13:29
In the last 7 days
Keenan: Reseachers are 'in this together' to solve rare diseases Calgary Herald13:11 10-Apr-21
Agios A Buy For Rare Disease Focus, Stock Buyback Seeking Alpha21:29 9-Apr-21
Fulcrum Therapeutics Presents Published Structure of Investigational Small Molecule FTX-6058 at the American Chemical… GlobeNewswire (Press Release)18:24 9-Apr-21
Successful liver transplant for girl with rare disease SHINE12:49 9-Apr-21
CENTOGENE to Announce Full Year 2020 Financial Results on April 15, 2021 BioSpace12:14 8-Apr-21
Ministry of Health developing concept of medical care for rare disease patients Ukrinform10:54 8-Apr-21
Austin man prepares for 16th marathon raising money for rare disease in children CBS Austin00:57 8-Apr-21
Patient centricity vital to advancing rare-disease treatments: ICON Outsourcing-Pharma.com18:37 7-Apr-21
Acanthamoeba Keratitis, Leishmaniasis and Dry Nose discussed with Profounda’s CEO Todd MacLaughlan GlobeNewswire (Press Release)17:18 7-Apr-21
Rare disease policy a mockery of right to health, says Bengaluru advocacy group The New Indian Express15:10 7-Apr-21
Dedicated dad hopes to run down cure for a rare disease Outsourcing-Pharma.com15:03 7-Apr-21
Soldiers from Chicksands take on ultra marathon challenge for children with rare... Biggleswade Today, Bedfordshire14:47 7-Apr-21
Harmony Biosciences Launches Grant Program To Address Inequities In The Delivery Of Healthcare… PR Newswire (Press Release)13:17 7-Apr-21
Insmed Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4) PR Newswire (Press Release)13:17 7-Apr-21
Tessera Therapeutics Strengthens Leadership Team to Advance ‘Gene Writing’ Platform &… Business Wire (Press Release)12:02 7-Apr-21
Applications for Rare Disease Fund Ministry of Health Singapore (Press Release)08:41 7-Apr-21
Andersen-Tawil Syndrome Market Gains Ground with Government Incentives Towards Rare Disease Treatments: Fact.MR BioSpace08:23 7-Apr-21
Decentralized Studies For Rare Diseases Trial Site News01:30 7-Apr-21
Takeda's rare disease med Natpara faces long road back to market as manufacturing issues… FiercePharma17:01 6-Apr-21
MCI OneHealth Continues to Accelerate Precision Medicine and Technology Roadmap Through the Acquisition of Khure Health GlobeNewswire (Press Release)13:38 6-Apr-21
BIS Research Publishes Nine Market Intelligence Reports in Precision Medicine in the First… PR Newswire (Press Release)13:36 6-Apr-21
Seelos Therapeutics Announces a Strategic Device Partnership with AptarGroup, Inc. PR Newswire (Press Release)12:06 6-Apr-21
Alnylam publish successful results from Phase III study of ultra-rare disease Pharmafile11:53 6-Apr-21
DuchenneXchange Launches V2.0 As Rare Disease Community Seeks "Safe" Place To Discuss… PR Newswire (Press Release)11:01 6-Apr-21
Sprout Health Solutions and Takeda Explore Digital Health Tools in Rare Disease During Covid-19 PR Newswire (Press Release)10:14 6-Apr-21
2,400 people remain without rare disease drug as Takeda manufacturing woes continue ENDPOINTS21:30 5-Apr-21
PRA ‘toolkit’ pinpoints rare-disease clinical trial risks Outsourcing-Pharma.com17:36 5-Apr-21
Rare Disease Therapeutics, Inc. Announces New Expanded FDA Approved Indication for Antivenom… Business Wire (Press Release)14:47 5-Apr-21
Foundation for USP7 Related Diseases Announces Grant to Neurolentech in Search for Cure to Rare … PR Newswire (Press Release)14:03 5-Apr-21
n-Lorem Foundation Partners with Ultragenyx to Bolster Development of Personalized Medicines… Business Wire (Press Release)13:08 5-Apr-21
Myovant Sciences Appoints Lauren Merendino as Chief Commercial Officer GlobeNewswire (Press Release)12:01 5-Apr-21
A good start: On rare diseases and government support for treatment The Hindu02:59 5-Apr-21
In the last month
National Policy for Rare Diseases approved by health minister: 7 points Hindustan Times06:13 4-Apr-21
Jharkhand: Rare disease and rarer act of generosity The New Indian Express05:37 4-Apr-21
Union Health Minister approves National Policy for Rare Diseases AniNews.in22:39 3-Apr-21
Health Minister Harsh Vardhan approves national policy for rare diseases Livemint.com16:41 3-Apr-21
Health minister Vardhan approves National Policy for Rare Diseases, 2021 Hindustan Times16:39 3-Apr-21
Health Minister Harsh Vardhan approves National Policy for Rare Diseases, 2021 The Financial Express16:25 3-Apr-21
Morning Digest: India stands for revival of democracy in Myanmar; disquiet over govt.’s new policy for rare diseases,… The Hindu03:50 3-Apr-21
ProQR Announces Virtual Presentation at American Association for Pediatric Ophthalmology and Strabismus (AAPOS) 2021 GlobeNewswire (Press Release)21:20 2-Apr-21
Disquiet over govt.’s new policy for rare diseases The Hindu16:49 2-Apr-21
A new collaboration demonstrates how tech can help understand rare diseases TechRepublic13:57 2-Apr-21
Patients of rare disease to get treatment under Ayushman Bharat Livemint.com07:59 2-Apr-21
Eloxx Acquires Zikani with Hopes to Rule the RNA Wave for Rare Disease BioSpace18:03 1-Apr-21
Toddler's life hangs in balance as drug for rare disease is set to be discontinued Bring Me The News16:37 1-Apr-21
Eloxx Pharmaceuticals Acquires Zikani Therapeutics GlobeNewswire (Press Release)12:56 1-Apr-21
Dad, 25, with rare disease desperate to save his sight so he can see his children grow up Wales Online11:16 1-Apr-21
India notifies first policy on rare diseases The Economic Times04:24 1-Apr-21
Otsuka exec on what's next for kidney care innovation and a peek at what's coming down the pipeline MobiHealthNews20:32 31-Mar-21
Rare disease in bears may make them appear friendly to humans USA Today EU16:44 31-Mar-21
Software engineer bets on technology to help speed rare disease treatments Science Magazine16:14 31-Mar-21
Uplifting Athletes Presents $140,000 in Rare Disease Research Grants at 2021 Young Investigator … PRWeb (Press Release)14:53 31-Mar-21
Rapid Genome Sequencing Can Save Babies With Rare Diseases, If They Can Get It Forbes12:03 31-Mar-21
Alexion’s Camilla Harder Hartvig on making the impossible possible in rare disease pharmaphorum11:58 31-Mar-21
Amryt Submits a New Drug Application to the US Food and Drug Administration for Oleogel-S10* (Filsuvez®) GlobeNewswire (Press Release)07:03 31-Mar-21
Cristiano Ronaldo's Castaway Armband Put On Auction To Help Baby Suffering From Rare Disease NDTV.com15:51 30-Mar-21
Irish boy with rare disease celebrates 10th birthday with messages from famous stars Irish Mirror15:27 30-Mar-21
Soligenix Announces Recent Accomplishments And Year-End 2020 Financial Results BioSpace13:17 30-Mar-21
Warning issued after healthy dog died from rare disease spread through rat urine Manchester Evening News19:27 29-Mar-21
Rare-disease researchers face unique obstacles: Minoryx Outsourcing-Pharma.com17:11 29-Mar-21
Ionis unveils positive PhII data for in-house rare disease drug, paving way to blockbuster rivalry with Takeda ENDPOINTS16:03 29-Mar-21
'A smile that would light up any room' - tributes pour in after Irish toddler tragically dies… Irish Mirror15:51 29-Mar-21
Report Lauds Orphan Drug Act But Notes Most Therapies Are Blocked From Competition American Journal of Managed Care02:47 28-Mar-21
‘Heartening’ support from CRDN to family: life with a rare disease for Tristan, 10 Cambridge Independent13:07 27-Mar-21
Patient with rare disease treated successfully in city hospital The Times of India23:14 26-Mar-21
Report: Orphan Drug Act Boosted Rare Disease Therapies, But Access is Uneven BioSpace16:22 26-Mar-21
Incurable Leigh Syndrome: Scientists create first human model for rare disease Medical Xpress16:19 26-Mar-21
Incurable Leigh Syndrome: German scientists create first human model for rare disease EurekAlert!14:01 26-Mar-21
Genome Sequencing Producing Precise Diagnoses for Range of Rare Diseases American Journal of Managed Care13:32 26-Mar-21
Global Orphan Drug Rare Disease Market Insight 2026 PR Newswire (Press Release)12:46 26-Mar-21
China At Global Rare Disease Crossroads: CanBridge CEO Scrip Pharma Intelligence08:08 26-Mar-21
New Study Investigates the Number of Available Orphan Products, Generics and Biosimilars PR Newswire (Press Release)13:30 25-Mar-21
FDNA Telehealth Brings A Fresh Global Perspective to Online Genetic Counseling Nature World News13:15 25-Mar-21
Cyclo Therapeutics Meets Primary Efficacy Endpoint in Phase 1/2 Trial from Intravenous… Business Wire (Press Release)12:36 25-Mar-21
Sangamo Therapeutics Announces Participation at Guggenheim Healthcare Talks: 2021 Genomic… Business Wire (Press Release)12:33 25-Mar-21
Magenta Therapeutics to Participate in the Guggenheim Healthcare Talks 2021 Genomic Medicines & … Business Wire (Press Release)12:03 25-Mar-21
Ionis shares tumble 19% after Roche halts Phase 3 study for rare-disease drug MarketWatch19:12 24-Mar-21
Horizon Therapeutics plc Launches The Horizon Prize with Massachusetts Institute of Technology… Business Wire (Press Release)12:01 24-Mar-21
From Hong Kong to Boston, New Biopharma Launches with Immune-based Pipeline BioSpace18:15 23-Mar-21
Shane Simpson heads into UVA Pro Day to raise awareness for special cause Wahoos24718:10 23-Mar-21
Notify health policy on rare diseases: HC The Hindu16:58 23-Mar-21
Bio Eats World: Solving Medical Mysteries in the World of Rare Disease Andreessen Horowitz15:25 23-Mar-21
PRA Health (PRAH) Unveils Toolkit for Rare Disease Patients Nasdaq14:16 23-Mar-21
Delhi HC gives nod for NCRD, orders to finalise National Health Poli AniNews.in13:46 23-Mar-21
A Precocious Teenager Faces a Rare Disease in Ae-ran Kim’s Touching Debut Novel, “My Brilliant Life” Words Without Borders12:32 23-Mar-21
Global Genes to Connect Rare Disease Patients and Families With Medical Students to Build a… Business Wire (Press Release)12:05 23-Mar-21
Ionis shares tumble19% after Roche halts Phase 3 study for rare-disease drug MarketWatch12:03 23-Mar-21
Recordati: Positive Results From the Phase III LINC-4 Study Presented at the Endocrine Society’s Annual Meeting… BioSpace11:26 23-Mar-21
Teenager dies from rare disease after mistaking swelling for allergic reaction Mirror.co.uk10:44 23-Mar-21
Notify health policy for rare diseases by March 31, HC asks Centre; directs setting up of R&D body The New Indian Express09:50 23-Mar-21
Zenas BioPharma Launches as a Cross-Border Biopharmaceutical Company to Bring Innovation to the Development and… AsiaOne08:16 23-Mar-21
Recordati Rare Diseases: Positive Results From the Phase III LINC 4 Study Presented Today at… Business Wire (Press Release)08:10 23-Mar-21
PRA Health Sciences toolkit to identify, mitigate rare disease risks Bernama07:27 23-Mar-21
PRA’s Center for Rare Diseases Launches Toolkit to Identify and Mitigate Risks to Rare Disease Clinical Programs GlobeNewswire (Press Release)20:04 22-Mar-21
Translate Bio Appoints Dr. Rand Sutherland as President GlobeNewswire (Press Release)11:34 22-Mar-21
Solving the “information challenge” of rare disease diagnosis pharmaphorum11:19 22-Mar-21
SpringWorks Therapeutics Appoints Bhavesh Ashar as Chief Commercial Officer BioSpace10:57 22-Mar-21
Genetic Counseling and Telehealth: a new virtual solution PRWeb (Press Release)04:09 22-Mar-21
Study finds impact of genome sequencing in diagnosing rare diseases AniNews.in09:26 21-Mar-21
Family's journey with rare disease highlights challenges 7News Denver03:48 20-Mar-21
Swiss rare disease biotech VectivBio Holding files for a $100 million US IPO Nasdaq00:13 20-Mar-21
Rare disease biotech Reneo Pharmaceuticals files for a $100 million IPO Nasdaq00:13 20-Mar-21
Saniona Chairman J. Donald deBethizy and CEO Rami Levin, as well as additional members of the board and management,… GlobeNewswire (Press Release)16:33 19-Mar-21
HRA Pharma debuts one-stop hub for rare disease Cushing’s syndrome aimed at physicians FiercePharma14:12 19-Mar-21
Gene Therapy Data Breathes New Life Into Battered Sarepta Stock Barron's Online12:28 19-Mar-21
Harmony Biosciences Hosts Summit Which Sheds Light on Impact of Sleep Disruption in Rare… PR Newswire (Press Release)12:08 19-Mar-21
Implementation of the UK Rare Diseases Framework The House of Commons Library blog10:24 19-Mar-21
New Advancements in Genetic Analysis For Rare Disease Delivering Accuracy & Affordability PRWeb (Press Release)04:12 19-Mar-21
More precise diagnoses made possible with whole genome sequencing ScienceDaily22:09 18-Mar-21
'Germ Hunters' discover rare disease in rural Alberta EurekAlert!20:04 18-Mar-21
Outlook on the Rare Disease Diagnostics Global Market to 2030 - Analysis and Forecast PR Newswire (Press Release)19:48 18-Mar-21
COVID's all-hands-on-deck approach should be standard for rare diseases The Hill16:44 18-Mar-21
Rare disease transmitted by bat and bird droppings discovered in rural Alberta Global News16:19 18-Mar-21
Local teenager makes macarons to help kids with rare diseases take a special ski trip CBS Austin15:06 18-Mar-21

      
            
      
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