News and Press Releases
PacBio and Radboud UMC Announce Remarkable Study Results Using HiFi Long Read Sequencing to Help Advance Rare Disease Diagnostics
14 January 2025 – Menlo Park, California US – PacBio (NASDAQ: PACB), a leading provider of highly accurate long-read sequencing solutions, announced a significant publication from Radboud University Medical Center...
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Posted: January 16, 2025
Johnson & Johnson MedTech Joins Experts to Define and Classify Surgical Site Outcomes
Healthcare experts call for consistent definitions in the assessment, reporting, and monitoring of surgical woundsA standardized classification system offers potential to capture and treat underreported surgical site infections and other...
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Posted: December 5, 2024
Novo Nordisk to present new data from sickle cell disease and haemophilia trials at the 66th American Society of Hematology (ASH) annual meeting
HIBISCUS phase 2 data evaluating the safety profile and efficacy of etavopivat in adult and adolescent patients with sickle cell disease FRONTIER4 interim phase 3 results evaluating safety profile and...
Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: December 3, 2024
PTC Therapeutics to Participate at Upcoming Investor Conferences
4 November 2024 -- New Jersey, US -- PTC Therapeutics, Inc today announced that the company will present a company overview at the following conferences: UBS Global Healthcare ConferenceWednesday, Nov....
Category: Biotechnology, Clinical Trials, Other, Pharmaceutical
Posted: November 4, 2024
PTC Therapeutics Inc. 100 Corporate Court South Plainfield, NJ 07080-2449
Nipocalimab demonstrates sustained disease control in adolescents living with generalised myasthenia gravis in the Phase 2/3 study
First FcRn blocker to demonstrate sustained disease control over 24 weeks in antibody positive adolescents aged 12 – 17 years, broadening the population in which nipocalimab has been studied 15...
Category: Clinical Trials, Drug Discovery, Pharmaceutical
Posted: October 15, 2024
Janssen-Cilag Limited 50-100 Holmers Farm Way High Wycombe Buckinghamshire HP12 4EG
Sterling Pharma Solutions Announces Partnership with Soligenix for the Manufacture of Oncology-Targeted API
3 October 2024 -- Cramlington, UK -- Sterling Pharma Solutions, a global contract development and manufacturing organisation, and Soligenix, a late-stage biopharmaceutical company focused on developing and commercialisation of products...
Category: BioManufacturing, Clinical Trials, Pharmaceutical
Posted: October 3, 2024
Sterling Place, Dudley Cramlington, Northumberland, NE23 7QG, United Kingdom
AzurBio Pharma Launches to Fast-Track European Market Entry for Biopharma Companies with Breakthrough Treatments
1 October 2024 -- Paris, France -- AzurBio Pharma, specializing in bringing innovative pharmaceutical products to European markets and focusing on rare and serious diseases with unmet medical needs, is...
Category: Biotechnology, Clinical Trials, Drug Discovery, Pharmaceutical
Posted: October 1, 2024
Sarepta Therapeutics Announces Recipients of LGMD Grant Awards for 2024; Program Supports Early Genetic Testing and Awareness
Recipients awarded a total of more than $100,000 in grants Funding will support education and activities to shorten diagnostic journey. 30 September 2024 -- Massachusetts, US -- Sarepta Therapeutics, Inc, the...
Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: September 30, 2024
Hill House, 1 Little New Street London, EC4A 3TR United Kingdom
Mogrify appoints Dr Jonathan Appleby as chief scientific officer
Strategic hire bolsters executive leadership team as company progresses multiple therapeutic programmes toward and into clinical development 15 July 2024 – Cambridge, UK – Mogrify, a regenerative medicine company developing...
Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: July 15, 2024
Biogen Receives European Commission Approval for QALSODY (tofersen), the First Therapy to Treat a Rare, Genetic Form of ALS
SOD1-ALS is a devastating, uniformly fatal and ultra-rare genetic form of ALS estimated to affect less than 1,000 people in Europe QALSODY is Biogen’s third rare disease therapy to be...
Category: Clinical Trials, Drug Discovery, Pharmaceutical
Posted: May 30, 2024
TMC Pharma champions holistic approach to medical services in rare disease drug development
13 March 2024 – In a recent webinar, rare disease specialist and global clinical research organisation (CRO) TMC Pharma gathered esteemed speakers to share insights into the scope of medical...
Category: Drug Discovery, Pharmaceutical
Posted: March 13, 2024
Lodge Farm Barn Elvetham Park Estate, Fleet Road, Hartley Wintney, Hampshire RG27 8AS, UK
Clinigen launches ‘What is Possible?’ campaign to accelerate access to medicines on Rare Disease Day
With over 7,000 rare diseases that impact over 300 million people globally, just 5% of rare diseases have a licensed treatment 29 February 2024 -- London, UK -- Clinigen, the...
Category: Other
Posted: February 29, 2024
