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Rare Diseases

In the last 6 hours

PacBio's Stock Up on Munster Deal for Male Infertility Research Zacks15:31

In the last 8 hours

Schoolboy, 11, 'ageing backwards' left with abilities of baby due to rare disease Daily Record13:14

Earlier today

Agios Announces FDA Orphan Drug Designation Granted to Tebapivat (AG-946) for Treatment of Myelodysplastic Syndromes… GlobeNewswire (Press Release)12:02

Oxford-Harrington Rare Disease Centre Appoints Influential UK Leader in Health Innovation Policy, Baroness Nicola… GlobeNewswire (Press Release)09:03

Yesterday

Viridian Therapeutics Announces Proposed Underwritten Public Offering of Common Stock and… Business Wire (Press Release)21:06 10-Sept-24

Big companies keep shuttering their co-branded credit cards. Why? Press-Telegram, California19:58 10-Sept-24

NIH Recognizes Yale’s Expertise in the Genetics of Rare Disease Yale School of Medicine19:36 10-Sept-24

FDA Grants Rare Pediatric Disease Designation to NS-050/NCNP-03 for the Treatment of Duchenne… PR Newswire (Press Release)16:00 10-Sept-24

RZLT Stock Up as FDA Lifts Clinical Holds on Rare Disease Study Zacks15:02 10-Sept-24

Rallybio to Present Results of Epidemiological Analysis Demonstrating FNAIT Risk Across… Business Wire (Press Release)13:09 10-Sept-24

Cornerstone Genomics Launches CodeXome: A Revolutionary Tool for Genetic Research & Rare… PRWeb (Press Release)13:08 10-Sept-24

Catawba mother donates her kidney to daughter diagnosed with rare disease WECT12:28 10-Sept-24

Genetic testing for rare diseases is hard to find if you live in rural Kansas, but now there's help Wichita Public Radio09:06 10-Sept-24

In the last 7 days

Catawba mother donates her kidney daughter diagnosed with rare disease WBTV23:11 9-Sept-24

Fierce Names Rallybio to the 2024 Fierce 50 List as an Innovation Honoree Business Wire (Press Release)22:20 9-Sept-24

Viridian Therapeutics to Webcast VRDN-001 Phase 3 THRIVE Topline Results on September 10, 2024 Business Wire (Press Release)21:03 9-Sept-24

FDA Lifts Partial Clinical Holds On Rezolute's Ersodetug To Treat Congenital hi; Stock Up RTT News13:36 9-Sept-24

Protara Therapeutics Announces Results from THRIVE-1, a Prospective Observational Study Evaluating the Prevalence of… GlobeNewswire (Press Release)13:08 9-Sept-24

Chiesi Global Rare Diseases Launches New Research Grant Initiative to Support Research… PR Newswire (Press Release)12:00 9-Sept-24

Kissei Partners with Reborna for Rare Disease Therapies TipRanks06:27 9-Sept-24

'A Night For Luca.' Patchogue rallies for 16-month-old boy diagnosed with rare diseases News 12 Long Island03:41 9-Sept-24

AI platform demonstrates promising results in treating patient with rare cancer The Hippocratic Post12:30 8-Sept-24

Artificial intelligence platform demonstrates promising results in effectively treating a… ScienceDaily22:10 7-Sept-24

There Was No Treatment for His Son’s Rare Disease, so Dad Moves Mountains to Make One for Children Worldwide Good News Network16:44 6-Sept-24

Mum's warning as her baby could lose sight in one eye after contracting rare disease at 10… Essex Live12:25 6-Sept-24

The rising demand for lentiviral vectors for in vivo gene therapy Drug Target Review10:07 6-Sept-24

Viridian Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) Business Wire (Press Release)21:08 5-Sept-24

RARE Navigator Pilot Program Aims to Eliminate Critical Gaps in Support for Patients and… PRWeb (Press Release)17:22 5-Sept-24

Victoria's Parliament Buildings set to glow red to shed light on rare disease Sooke News Mirror15:20 5-Sept-24

Artificial intelligence platform demonstrates promising results in effectively treating a… ecancer14:16 5-Sept-24

Sarepta Therapeutics Announces Recipients of the 7th Annual Route 79, The Duchenne Scholarship… Business Wire (Press Release)14:01 5-Sept-24

Agios to Present at the 2024 Cantor Global Healthcare Conference on September 18, 2024 GlobeNewswire (Press Release)12:02 5-Sept-24

Cures for Rare Diseases Now Exist. Employers Don't Want to Pay BNN Bloomberg11:34 5-Sept-24

European Commission grants orphan drug designation for rare disease gene therapy Biopharma Reporter10:53 5-Sept-24

A rare disease, without a cure: September is Sickle Cell Anemia Awareness Month WCTV23:52 4-Sept-24

Parents fight for a cure, plus viral outbreaks and benefits of weekend sleep Fox News23:46 4-Sept-24

Rare Disease-Focused Ascendis Pharma Stock Falls On Underwhelming Q2 Earnings, Guidance Cut Benzinga14:19 4-Sept-24

Revio to Power Research in Male Infertility and Rare Disease at Münster University Hospital GlobeNewswire (Press Release)14:07 4-Sept-24

We are running for Ruby… little Lancaster girl with rare disease inspires Great North Run… Lancaster Guardian12:05 4-Sept-24

Patients with a rare kidney disease to get financial assistance for transplants Mint11:22 4-Sept-24

The Dion Foundation and Atamyo Therapeutics Announce a Partnership to Expand into the US… Business Wire (Press Release)10:33 4-Sept-24

In the last month

RegenxBio touts positive data for rare disease drug RGX-121 Pharmaceutical Technology23:10 3-Sept-24

Quince Therapeutics to Present at H.C. Wainwright 26th Annual Global Investment Conference Business Wire (Press Release)21:08 3-Sept-24

Father Creates a New Drug To Save His Son From Rare Disease The New York Sun20:04 3-Sept-24

Spain co-sponsors WHO resolution on rare diseases to improve their diagnosis, treatment and care Government of Spain (Press Release)14:20 3-Sept-24

Eastbourne boy, 2, with rare disease to take part in charity danceathon SussexWorld13:08 3-Sept-24

PANTHERx® Rare Selected by Calliditas Therapeutics as Specialty Pharmacy for TARPEYO® PR Newswire (Press Release)13:05 3-Sept-24

Arrowhead to file NDA after pivotal rare disease trial meets all endpoints Clinical Trials Arena11:40 3-Sept-24

A framework for sharing of clinical and genetic data for precision medicine applications Nature.com11:07 3-Sept-24

Father created a drug to save his son from a rare disease, now other families are desperate to… Fox News12:14 2-Sept-24

Harnessing AI for the unseen: A call to action for rare diseases like glossopharyngeal neuralgia The New Times08:56 2-Sept-24

Relief Therapeutics Announces PKU GOLIKE Study Results Presentation at SSIEM 2024 DGAP06:03 2-Sept-24

4-year-old battles rare disease months after it killed his 2-year-old brother, family says KCRG TV 9, Iowa20:34 1-Sept-24

Rare disease China ‘doctor in a wheelchair’ from top university, inspires students South China Morning Post07:05 1-Sept-24

Sarepta Therapeutics Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) Business Wire (Press Release)21:18 30-Aug-24

ESC Congress: Amvuttra data signals positive prospects in cardiomyopathy for Alnylam Pharmaceutical Technology19:43 30-Aug-24

At 13.1% CAGR, Rare Diseases Market Size Anticipated to Reach USD 547.5 Billion by 2030: Prophecy Market Insights GlobeNewswire (Press Release)14:22 30-Aug-24

Sarepta Therapeutics to Present at the Morgan Stanley 22nd Annual Global Healthcare Conference Business Wire (Press Release)13:31 30-Aug-24

Powell 4-year-old battles rare disease, months after it kills his brother WVLT03:22 30-Aug-24

Poseida Therapeutics to Present at Upcoming Investor Conferences PR Newswire (Press Release)21:06 29-Aug-24

Discovery gives answers to parents of children with rare disease ScienceDaily18:36 29-Aug-24

Inozyme Pharma to Participate at the 2024 Wells Fargo Healthcare Conference GlobeNewswire (Press Release)13:36 29-Aug-24

Tandem challenge to fundraise for ultra-rare disease affecting 70 worldwide, including two siblings Strathspey and Badenoch Herald, Scotland13:08 29-Aug-24

Rallybio Announces Publication of RLYB212 Phase 1 Proof-of-Concept Study Results in Thrombosis… Business Wire (Press Release)13:06 29-Aug-24

Stittsville wheelchair fencer set to compete at Paralympics Ottawa Sun09:15 29-Aug-24

JCR Pharmaceuticals Advances on Rare Disease Trial TipRanks08:37 29-Aug-24

Bismarck attorney writes children’s book for son with rare disease KMOT00:17 29-Aug-24

New Hampshire Man Dies After Mosquito Bite Transmits Rare Disease Missoulian, Montana18:41 28-Aug-24

PsychoGenics Inc. Awarded $3 Million SBIR Grant to Employ Its AI-Enabled Platforms to Find… Business Wire (Press Release)13:02 28-Aug-24

FDA Grants Priority Review to SpringWorks Therapeutics’ New Drug Application for Mirdametinib for the Treatment of… GlobeNewswire (Press Release)11:34 28-Aug-24

RARE-NL: LUMC joins consortium to find treatments for rare diseases Leiden University08:48 28-Aug-24

US Man Dies From Rare Disease Following Mosquito Bite ScienceAlert03:36 28-Aug-24

Fulcrum Therapeutics to Participate in Upcoming September Conferences GlobeNewswire (Press Release)21:08 27-Aug-24

Former Regenxbio leaders take rare disease gene therapies to startup with $15M ENDPOINTS15:32 27-Aug-24

North America Rare Disease Therapeutics Market Size, Trends, Competition, Forecasts & Opportunities, 2019-2023 &… GlobeNewswire (Press Release)14:32 27-Aug-24

Rallybio to Present at Upcoming Investor Conferences Business Wire (Press Release)13:04 27-Aug-24

Groundbreaking Study by RDDC and NORD: Unveiling Inequities in the Rare Disease Community… PR Newswire (Press Release)13:01 27-Aug-24

Diaceutics reports progress with rare-disease offerings ShareCast12:24 27-Aug-24

Enhanced Diaceutics offering to “revolutionise” early identification of patients Insider Media08:36 27-Aug-24

Diaceutics Boosts Rare-Disease Services and Secures New Contract TipRanks07:48 27-Aug-24

Avanzanite Bioscience Reports European Commission's Authorisation of Orphan Medicinal Product… Business Wire (Press Release)06:01 27-Aug-24

Dr. Fauci Hospitalized With Rare Disease Futurism19:10 26-Aug-24

Rare Disease Patient to Walk from Los Angeles to San Diego to Raise Awareness of Condition PRWeb (Press Release)12:07 26-Aug-24

A rare disease changed Archie’s life. Fruit flies are now his best hope WA Today07:01 25-Aug-24

Global Genes' Annual Week in RARE Event Heading to Kansas City PRWeb (Press Release)21:48 22-Aug-24

Eton Pharmaceuticals to Participate at the H.C. Wainwright 26th Annual Global Investment Conference GlobeNewswire (Press Release)21:37 22-Aug-24

While FDA mulls Zevra's rare disease med, Rafael merges with Cyclo to push their rival candidate FierceBiotech14:02 22-Aug-24

By 2034, Pediatric Clinical Trials Market Size & Share to Reach USD 56.2 Billion, at 6.6% CAGR: Prophecy Market Insights GlobeNewswire (Press Release)12:43 22-Aug-24

Rare disease drug from LEO's Timber felled by failed study pharmaphorum10:49 22-Aug-24

STAT Plus: Gene therapy pioneer Jim Wilson believes he can commercialize rare-disease drugs in… STAT09:35 22-Aug-24

Family of young Edmonton boy born with rare disease has hope after stem cell transplant CTV News Edmonton00:46 22-Aug-24

PAPA can you hear me? BioTek reMEDys in rare disease awareness push FiercePharma16:38 21-Aug-24

Pyros Pharmaceuticals’ Abstract Accepted for Poster Presentation at the American Epilepsy… Business Wire (Press Release)21:06 20-Aug-24

BioTek reMEDys Promotes Awareness and Education for Autoinflammatory Conditions Business Wire (Press Release)17:52 20-Aug-24

Taking the Pulse, A Health Care and Life Sciences Video Podcast | Episode 203: Manufacturing Specialty Drugs for Rare… JD Supra17:12 20-Aug-24

Roche regains reputation crown among rare disease patient groups, with Amgen close behind FiercePharma16:35 20-Aug-24

Ayvakit's explosive 185% revenue growth: A game-changer in rare disease treatment Outsourcing-Pharma.com16:30 20-Aug-24

Upsher-Smith Laboratories, LLC Selects PANTHERx® Rare for the Distribution of TORPENZ™… PR Newswire (Press Release)14:43 20-Aug-24

Ambry Genetics Announces New Multiomic Exome Test for Improved Rare Disease Detection Business Wire (Press Release)14:03 19-Aug-24

Q&A: Lilly’s top scientists on genetic medicine ambitions, and how they’re not just for rare diseases ENDPOINTS12:23 19-Aug-24

Fulcrum Therapeutics Strengthens Leadership Team with the Appointments of Isabel Kalofonos as Chief Commercial Officer… GlobeNewswire (Press Release)12:04 19-Aug-24

Rare diseases point to metabolism-immunity link Gulf News11:40 18-Aug-24

Recognise Huntington’s Disease as a rare ailment, urge HD patients and their caregivers The Hindu02:36 17-Aug-24

Child with ultra-rare disease gets a treatment just for her Nature.com21:36 16-Aug-24

Rare diseases point to connections between metabolism and immunity Newswise (Press Release)19:04 16-Aug-24

PANTHERx® Rare Partnering with Gilead Sciences, Inc. for the Distribution of LIVDELZI® PR Newswire (Press Release)16:38 16-Aug-24

Amicus Therapeutics: A Compelling Investment In The Rare Disease Biotech Sector Seeking Alpha08:37 16-Aug-24

AHN Becomes First Health Network in Western Pennsylvania to Utilize New Data Tool to Identify… PRWeb (Press Release)14:38 15-Aug-24

Soleno Therapeutics Announces Updates to its Board of Directors GlobeNewswire (Press Release)13:01 15-Aug-24

15-year-old girl with rare disease stores her eggs in Shanghai SHINE09:56 15-Aug-24

European Medicines Agency Confirms Acceptance of Marketing Application for AVT06, a Proposed Biosimilar to Eylea®… GlobeNewswire (Press Release)09:30 15-Aug-24

Men's cancer warnings, rare diseases revealed, and babies' chiropractic care Fox News02:20 15-Aug-24

Family of toddler who died of rare disease hope for test changes to avoid needless deaths Sunday World15:33 14-Aug-24

Petauri™ Acquires Medical Communications Agencies in Major Expansion PR Newswire (Press Release)13:00 14-Aug-24

Mayo family raise funds for child with rare disease The Mayo News10:34 14-Aug-24

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11 Sept 15:31

About our Rare Diseases news

NewsNow brings you the latest news from the world’s most trusted sources on Rare Diseases. NewsNow aims to be the world’s most accurate and comprehensive Rare Disease news aggregator, bringing you the latest headlines automatically and continuously 24/7.

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