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News and Press Releases

PTC Therapeutics Announces FDA Acceptance for Filing of NDA for Sepiapterin for the Treatment of Pediatric and Adult Phenylketonuria Patients

1 October 2024 -- New Jersey, US -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of...

Category: Biotechnology, Drug Discovery, Pharmaceutical
Posted: October 1, 2024

PTC Therapeutics
info@ptcbio.com
PTC Therapeutics Inc. 100 Corporate Court South Plainfield, NJ 07080-2449

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

AzurBio Pharma
32 Av. Kléber, 75116 Paris, France

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

Sarepta Therapeutics
tsorrentino@sarepta.com
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

Mogrify
info@mogrify.co.uk
25 Cambridge Science Park Milton Rd, Milton, Cambridge CB4 0FW

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

Biogen
Biogen International GmbH Neuhofstrasse 30 6340 Baar Switzerland

Successful Conclusion of London Biotechnology Show, Leaves Attendees Highly Satisfied

10 May 2024 -- London, UK -- The curtain has closed on the debut London Biotechnology Show 2024, leaving attendees thoroughly impressed by the seamless delivery of promised content and...

Category: Biotechnology
Posted: May 10, 2024

London Biotechnology Show
info@londonbiotechshow.com

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

TMC Pharma
info@tmcpharma.com
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

Clinigen
25 Bedford Square Bloomsbury London WC1B 3HH