Yesterday
X4 Pharmaceuticals Announces Presentation of Additional Data from Mavorixafor Phase 2 Trial in Chronic Neutropenia at… GlobeNewswire (Press Release)12:04 9-Dec-23
Rallybio Presents Natural History Study for Fetal and Neonatal Alloimmune Thrombocytopenia at… Business Wire (Press Release)12:02 9-Dec-23
In the last 7 days
SpringWorks Therapeutics Announces Closing of Upsized Public Offering of Common Stock and Full Exercise of… GlobeNewswire (Press Release)16:09 8-Dec-23
Chutes & Ladders—BIO lands rare disease advocate John Crowley as CEO FierceBiotech09:30 8-Dec-23
Teen's song in memory of cousins who died from rare disease released for Christmas The Northern Echo04:20 8-Dec-23
Whole Exome Sequencing Advancements in Unraveling Rare Diseases in Japan AsiaOne02:10 8-Dec-23
Heartbroken parents say girl, 7, fading before their eyes due to rare childhood dementia The Mirror02:03 8-Dec-23
Young Aberdeenshire gymnast struck with rare disease hails new bone marrow report Aberdeen Live01:44 8-Dec-23
New BIO Poll Reveals Americans Strongly Support the Orphan Cures Act Biotechnology Innovation Organization (Press Release)13:57 7-Dec-23
Reps. Eshoo and McCaul Introduce Bill to Strengthen Development of Pediatric Drugs Congresswoman Anna Eshoo (Press Release)12:49 7-Dec-23
Decentralized Clinical Trials: Diversity in Clinical Trials JD Supra11:28 7-Dec-23
University Hospital in Varna Joins European Programme for Patients with Rare Diseases Bulgarian News Agency11:20 7-Dec-23
Lobe Sciences Provides Update on L-130 Clinical Program Business Wire (Press Release)07:05 7-Dec-23
Congress Incentivizes Rare Disease Research. Big Pharma Exploits It. Mother Jones06:22 7-Dec-23
100-hour channel underway to raise funds for rare disease research My Algoma Manitoulin Now06:11 7-Dec-23
RARE-X Launches Pompe Consortium to Support Patient-Owned Data Collection and Aggregated Data… PRWeb (Press Release)14:00 6-Dec-23
Here’s What I Wish You Knew About My Rare Disease Prevention.com11:39 6-Dec-23
Manchester research leads to NHS roll out of life-saving treatment for babies with rare disease Manchester University NHS Foundation Trust (Press Release)11:11 6-Dec-23
Rare disease health education competition launched in Shanghai SHINE07:16 6-Dec-23
Establishment of National Rare Disease Steering Group Anglo Celt06:34 6-Dec-23
New steering group for rare diseases to be set up as patients waiting up to five years for… The Irish Independent05:28 6-Dec-23
FDA Roundup: December 05, 2023 PR Newswire (Press Release)17:38 5-Dec-23
FDA Roundup: December 5, 2023 U.S. Food and Drug Administration (Press Release)17:03 5-Dec-23
Call to publicize drug reviews must be discussed: NHIA Taipei Times10:41 5-Dec-23
Insmed Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4) - December 5, 2023 BioSpace09:49 5-Dec-23
STAT Plus: BIO selects rare disease advocate, biotech executive John Crowley as new CEO STAT09:49 5-Dec-23
Getting personal: a patient-centred approach to rare disease drug development Manufacturing Chemist06:47 5-Dec-23
Lobe Sciences Provides Update on L-130 Oral Psilocin Compound Business Wire (Press Release)06:36 5-Dec-23
Amicus Therapeutics Congratulates Executive Chairman John F. Crowley on Appointment as Next President and CEO of BIO GlobeNewswire (Press Release)06:04 5-Dec-23
Relief Therapeutics Shifts Commercial Operations to Partnership Model, Focusing on Efficient Patient Access and R&D… BioSpace04:04 5-Dec-23
SpringWorks Therapeutics Announces Pricing of Upsized Public Offering of Common Stock - December 05, 2023 BioSpace01:11 5-Dec-23
X4 Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) - December 04, 2023 BioSpace00:41 5-Dec-23
SpringWorks Therapeutics Announces Pricing of Upsized Public Offering of Common Stock GlobeNewswire (Press Release)00:38 5-Dec-23
The RNA Therapeutics Race: How Can Pharma Stay Ahead Of The Wave? Drugs Discovery Online00:05 5-Dec-23
STAT Plus: Official: FDA needs staff influx to meet gene therapy needs STAT16:40 4-Dec-23
SpringWorks Therapeutics Announces Proposed Public Offering of Common Stock GlobeNewswire (Press Release)16:14 4-Dec-23
Virginia girl, 8, is suffering 'childhood dementia' as her body slowly shuts down from rare… Mail Online13:13 4-Dec-23
Health experts insist that NHI benefits should include rare diseases News2411:08 4-Dec-23
Plymouth mum needs lifesaving treatment after cancer and rare disease diagnoses Plymouth Live09:15 4-Dec-23
Woodbury native diagnosed with rare disease turns tragedy into strength by writing TV pilot News 12 Long Island08:48 3-Dec-23
In the last month
Esquimalt family spreading kindness and fighting for cure to rare disease that claimed daughter’s life CHEK News19:38 2-Dec-23
Seelos Therapeutics Announces Closing of Public Offering PR Newswire (Press Release)16:07 1-Dec-23
7-year-old with rare disease enjoys holidays for first time thanks to new medication CBS Philadelphia19:54 30-Nov-23
Observe Sept 7 as a day to raise awareness on DMD disease: Kuwait Kuwait Times12:12 30-Nov-23
Benefit to be held Saturday in Wausau for toddler with rare disease WSAW11:54 30-Nov-23
Charles River works on rare disease gene therapy; Emergent gets $75M for anthrax vaccine; EuroAPI hires new COO ENDPOINTS11:39 30-Nov-23
Protara Therapeutics Announces Presentation of Additional Encouraging Data from Phase 1a ADVANCED-1 Trial of TARA-002… GlobeNewswire (Press Release)08:58 30-Nov-23
QPS Holdings, LLC divests its Neuropharmacology Business Unit Business Wire (Press Release)08:21 30-Nov-23
Scantox acquires QPS Austria's Neuropharmacology Division PR Newswire (Press Release)08:01 30-Nov-23
Teen releases song in tribute to young relatives who died of rare disease The Telegraph & Argus06:51 30-Nov-23
Project CASK Launches Ultra RARE Art to Cure an Ultra Rare Disease Little Black Book05:26 30-Nov-23
mRNA therapies: a new frontier European Pharmaceutical Review04:05 30-Nov-23
German researchers welcome national, EU support for gene therapy research EurActiv.com03:09 30-Nov-23
Farringdon owner’s warning as rare disease kills dog Liphook Herald12:22 29-Nov-23
Determined Austin mom takes on rare disease: Launches fundraiser to seek potential treatment for son's debilitating… CBS Austin10:51 29-Nov-23
Rare disease patients across Spain don’t have the same access to treatments EurActiv.com09:32 29-Nov-23
Insmed To Present at the Nasdaq 49th Investor Conference PR Newswire (Press Release)08:02 29-Nov-23
AstraZeneca advances scientific leadership in hematology at ASH 2023 Business Wire (Press Release)07:04 29-Nov-23
Mum raising awareness of twins' rare disease BBC05:27 29-Nov-23
Kyowa Kirin Responds to NICE’s Publication of Appraisal Consultation Document for CRYSVITA®… Business Wire (Press Release)03:10 29-Nov-23
OASYS NOW launches GRIP: aims to revolutionise treatment discovery for chronic and rare diseases Silicon Canals02:57 29-Nov-23
Seelos Therapeutics Announces Pricing of $5.55 Million Public Offering PR Newswire (Press Release)21:27 28-Nov-23
Seelos Therapeutics Announces Proposed Public Offering PR Newswire (Press Release)16:11 28-Nov-23
Takeda aligns with leading rare disease group for calls to action around health equity diagnoses ENDPOINTS10:50 28-Nov-23
Charles River and Genetic Cures for Kids Announce Gene Therapy Manufacturing Collaboration Business Wire (Press Release)08:10 28-Nov-23
Mum who lost daughter to rare disease pays tribute to 'warrior princess' Irish Mirror06:40 28-Nov-23
Calliditas Therapeutics: Undervalued Rare Disease Commercial Stage Biotech (Maintaining Buy) Seeking Alpha18:40 27-Nov-23
SpringWorks Therapeutics Announces FDA Approval of OGSIVEO™ (nirogacestat) as the First and Only Treatment for Adults… GlobeNewswire (Press Release)18:05 27-Nov-23
Marks: FDA open to using single-arm studies for accelerated approval of rare disease gene therapies ENDPOINTS15:22 27-Nov-23
8 hockey teams across CO help 4-year-old get treatment for rare disease 7News Denver14:42 27-Nov-23
Pro Research: Wall Street weighs in on Alnylam's future Investing.com12:50 27-Nov-23
Cost of Exempting Sole Orphan Drugs From Medicare Negotiation Journal of American Medical Associations11:54 27-Nov-23
External controls can augment low populations in rare disease trials Verdict Medical Devices10:50 27-Nov-23
First treatment recommended for infants with Wolman Disease European Pharmaceutical Review09:29 27-Nov-23
Amazing Manchester research leads to life-saving treatment for babies with rare disease I Love Manchester08:47 27-Nov-23
Arcturus Therapeutics Receives Orphan Drug Designation From The U.S. FDA For ARCT-032, For The… Drugs Discovery Online08:00 27-Nov-23
AstraZeneca's rare disease unit receives NICE recommendation for Wolman disease therapy in infants PMLiVE07:27 27-Nov-23
Mesoblast Files for Orphan Drug and Pediatric Rare Disease Designations for Rexlemestrocel-L as Treatment for Severe… GlobeNewswire (Press Release)19:01 26-Nov-23
Study shows how to treat hereditary, rare diseases AniNews.in04:37 26-Nov-23
'Made in India' drugs to cut rare disease treatment costs by nearly 100 times Wio News01:09 26-Nov-23
Boy, 9, Who's Losing Eyesight Takes Life’s Lemons and Makes Lemonade Inside Edition10:11 25-Nov-23
Oxford-Harrington Rare Disease Centre named in first global initiative for children with rare diseases University of Oxford04:13 25-Nov-23
India-made drugs reduce treatment costs for rare diseases by 100-fold NewsBytes02:29 25-Nov-23
Medicines for four rare diseases become cheaper due to domestic production The Times of India01:24 25-Nov-23
'Made in India' Drugs For 4 Rare Diseases Reduce Treatment Cost By Nearly 100 Folds News18.com22:39 24-Nov-23
Cheaper Made-in-India drugsto treat extremely rare diseases Hindustan Times20:20 24-Nov-23
Government approves 4 home-grown, cheaper drugs for rare diseases The Times of India18:38 24-Nov-23
Major drop in cost of drugs for 4 rare diseases after Indian companies begin production Indian Express17:43 24-Nov-23
For the first time, ‘Made in India’ drugs for rare diseases were launched Nord News13:16 24-Nov-23
In a first, 'Made in India' drugs launched for rare diseases Hindustan Times12:41 24-Nov-23
Cost-effective drugs for 14 rare diseases, including sickle cell, to be launched soon: Mansukh Mandaviya The New Indian Express12:31 24-Nov-23
For the first time, 'Made in India' drugs launched for rare diseases AniNews.in12:02 24-Nov-23
Indian Medicines For 4 Rare Diseases Cut Treatment Cost By Nearly 100 Times NDTV.com11:21 24-Nov-23
Four generic, made-in-India drugs to treat rare diseases offer relief for patients; more in pipeline The Hindu11:13 24-Nov-23
Seelos Therapeutics Announces 1-for-30 Reverse Stock Split PR Newswire (Press Release)10:56 24-Nov-23
Keeping rare disease patients at the heart of the EU’s review of the pharmaceutical legislation The Parliament Magazine06:53 24-Nov-23
Genprex gains FDA orphan drug status for lung cancer treatment Investing.com09:28 23-Nov-23
Expanding Horizons: Launching a Rare Disease Nonprofit Amid Government Funding Challenges American Journal of Managed Care08:11 23-Nov-23
Statistics on Rare Diseases Fund Application and Awareness of Donor Options Ministry of Health Singapore (Press Release)04:13 23-Nov-23
Rare Disease Fund Ministry of Health Singapore (Press Release)04:13 23-Nov-23
Biotech companies struggle for funds Northwest Arkansas Democrat-Gazette03:50 23-Nov-23
A Biotech Slump Is Emptying Labs and Threatening Research on New Drugs BNN Bloomberg06:55 22-Nov-23
Relief Therapeutics Announces CEO Transition BioSpace04:01 22-Nov-23
FDA Grants Orphan Drug Designation To NeoImmuneTech's NT-I7 For The Treatment Of Acute… Drugs Discovery Online01:24 22-Nov-23
Skip Therapeutics and Sheba Medical Center to Collaborate for the Development of RNA-based Treatments for Rare Diseases BioSpace23:47 21-Nov-23
Habs Stanley Cup winner, superfan join forces to support Montreal kid with rare disease CityNews Montreal15:35 21-Nov-23
Merck to Pay Up to $610 Million for Rare-Disease Therapies (1) Bloomberg Law14:05 21-Nov-23
BioTek reMEDys Urges Patients to Be Aware of Open Enrollment Deadlines Business Wire (Press Release)12:16 21-Nov-23
Brave mother donates part of her liver to to save son's life after he was diagnosed with a rare … Mail Online10:43 21-Nov-23
MSD reaches $610m deal to buy lysosome biotech Caraway pharmaphorum10:05 21-Nov-23
Merck to Pay Up to $610 Million for Rare-Disease Drug Developer BNN Bloomberg08:12 21-Nov-23
Viridian Therapeutics to Participate in 6th Annual Evercore ISI HealthCONx Conference Business Wire (Press Release)08:02 21-Nov-23
Zevra Therapeutics completes Acer acquisition in $91m deal Pharmaceutical Technology03:47 21-Nov-23
mRNA Advances Allow Drug Development Focus on Rare Diseases (Part Two) PharmTech.com16:06 20-Nov-23
mRNA Advances Allow Drug Development Focus on Rare Diseases (Part One) PharmTech.com15:25 20-Nov-23

      
            
      
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