Rare Diseases

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In the last half-hour

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

In the last 2 hours

RZLT Stock Up as FDA Lifts Clinical Holds on Rare Disease Study Zacks15:02

In the last 4 hours

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

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

In the last 6 hours

Catawba mother donates her kidney to daughter diagnosed with rare disease WECT12:28

In the last 8 hours

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

Yesterday

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

In the last 7 days

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

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

In the last month

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

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10 Sept 16:00

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