https://www.epmmagazine.com/topics/rare-diseases/index.rss en-gb © Rapid Life Sciences Ltd, a Rapid News Group Company. All Rights Reserved. Wed, 10 Feb 2021 09:50:00 -0000 Ipsen Pharmaceuticals and rare disease digital health specialists, Mendelian, has launched a new partnership to utilise Mendelian’s MendelScan software, a Class 1 Medical Device capable of aiding the rapid identification of people with rare diseases. https://www.epmmagazine.com/pharma-manufacturing-news/digital-health-in-pharma-news/ipsen-forms-digital-health-partnership-to-boost-rare-disease/ https://www.epmmagazine.com/pharma-manufacturing-news/digital-health-in-pharma-news/ipsen-forms-digital-health-partnership-to-boost-rare-disease/ Wed, 10 Feb 2021 09:50:00 -0000 Rare disease Life sciences consultancy RBW Consulting has announced it is funding a clinical research project in partnership with Rare Disease Research Partners https://www.epmmagazine.com/pharma-manufacturing-news/latest-pharmaceutial-news/new-research-announced-into-mental-health-impact-of-rare-dis/ https://www.epmmagazine.com/pharma-manufacturing-news/latest-pharmaceutial-news/new-research-announced-into-mental-health-impact-of-rare-dis/ Thu, 21 Jan 2021 12:18:41 -0000 Mental Health Italian pharmaceutical firm Chiesi has launched a new division focused on research and development initiatives for rare and ultra-rare diseases. https://www.epmmagazine.com/pharma-manufacturing-news/chiesi-launches-rare-disease-division/ https://www.epmmagazine.com/pharma-manufacturing-news/chiesi-launches-rare-disease-division/ Mon, 17 Feb 2020 15:10:00 -0000 Rare disease A new ‘game-changing’ DNA test on the NHS is said to be able to rapidly diagnose rare diseases for critically ill babes and children. https://www.epmmagazine.com/pharma-manufacturing-news/dna-test-t/ https://www.epmmagazine.com/pharma-manufacturing-news/dna-test-t/ Mon, 06 Jan 2020 12:00:00 -0000 Gene Technology AI technology company Healx has launched a Global Accelerator Programme for rare diseases to help discover new treatments. https://www.epmmagazine.com/pharma-manufacturing-news/healx-launches-accelerator-programme-for-rare-diseases/ https://www.epmmagazine.com/pharma-manufacturing-news/healx-launches-accelerator-programme-for-rare-diseases/ Thu, 17 Oct 2019 09:44:20 -0000 disease research A rare disease has been found to be more common than initially thought, according to a new report. https://www.epmmagazine.com/pharma-manufacturing-news/rare-disease-more-common-than-ini/ https://www.epmmagazine.com/pharma-manufacturing-news/rare-disease-more-common-than-ini/ Thu, 03 Oct 2019 08:38:16 -0000 Rare disease Emily Folk Emily Folk looks at the ways in which additive manufacturing could be implemented into pharmaceutical manufacturing processes. https://www.epmmagazine.com/pharmaceutical-industry-insights/the-potential-for-additive-manufacturing-in-pharmaceutical-p/ https://www.epmmagazine.com/pharmaceutical-industry-insights/the-potential-for-additive-manufacturing-in-pharmaceutical-p/ Thu, 28 Mar 2019 14:20:00 -0000 Additive manufacturing Peter Fish Dr Peter Fish, head of clinical partnerships at Mendelian discusses why artificial intelligence (AI) could be the key to diagnosing rare diseases. https://www.epmmagazine.com/pharmaceutical-industry-insights/why-ai-could-be-key-for-rare-diseases/ https://www.epmmagazine.com/pharmaceutical-industry-insights/why-ai-could-be-key-for-rare-diseases/ Mon, 25 Mar 2019 09:15:00 -0000 AI The government has announced a new National Genomic Healthcare Strategy to make the UK a world leader in preventing, diagnosing and treating people with rare diseases. https://www.epmmagazine.com/pharma-manufacturing-news/government-announces-new-genomics-strategy-on-rare-disease-d/ https://www.epmmagazine.com/pharma-manufacturing-news/government-announces-new-genomics-strategy-on-rare-disease-d/ Thu, 28 Feb 2019 15:55:00 -0000 Genomics Over the past 10 years, the diagnoses of rare diseases in patients has cost the NHS over £3.4 billion, new research shows. https://www.epmmagazine.com/pharma-manufacturing-news/study-shows-impact-of-rare-diseases-on-nhs/ https://www.epmmagazine.com/pharma-manufacturing-news/study-shows-impact-of-rare-diseases-on-nhs/ Tue, 18 Dec 2018 09:00:00 -0000 MRI A new funding scheme to help researchers better understand rare diseases has been announced by Great Ormond Street Hospital Children’s Charity (GOSH) and medical research charity LifeArc. https://www.epmmagazine.com/pharma-manufacturing-news/%C2%A31m-funding-scheme-for-rare-diseases-announced-by-go/ https://www.epmmagazine.com/pharma-manufacturing-news/%C2%A31m-funding-scheme-for-rare-diseases-announced-by-go/ Thu, 13 Dec 2018 09:35:00 -0000 Rare diseases The UK has become the first nation in the world to complete the largest genome sequencing project in healthcare, the government has announced. https://www.epmmagazine.com/pharmaceutical-industry-insights/uk-reaches/ https://www.epmmagazine.com/pharmaceutical-industry-insights/uk-reaches/ Thu, 06 Dec 2018 09:50:00 -0000 genome sequencing The US Food and Drug Administration (FDA) has granted accelerated approval for a new treatment for adults with Fabry disease — a rare genetic disorder, resulting from the build-up of globotriaoscylceramide (GL-3) in blood vessels and organs. https://www.epmmagazine.com/pharma-manufacturing-news/fda-grants-accelerated-approval-for-fabry-treatment/ https://www.epmmagazine.com/pharma-manufacturing-news/fda-grants-accelerated-approval-for-fabry-treatment/ Wed, 15 Aug 2018 13:18:50 -0000 approval Specialist British pharmaceutical wholesaler, Chapper healthcare, has published a white paper exploring the global challenges of access to and delivery of speciality medicines. https://www.epmmagazine.com/pharma-manufacturing-news/chapper-healthcare-publishes-white-paper-exploring-challenge/ https://www.epmmagazine.com/pharma-manufacturing-news/chapper-healthcare-publishes-white-paper-exploring-challenge/ Fri, 27 Apr 2018 11:10:32 -0000 International medicines research A new study from the University of Eastern Finland, published in the European Journal of Clinical Pharmacology, has highlighted that there are differences in the availability of orphan medicines between different European countries. https://www.epmmagazine.com/pharmaceutical-industry-insights/availability-of-orphan-medicines-varies-by-country-study/ https://www.epmmagazine.com/pharmaceutical-industry-insights/availability-of-orphan-medicines-varies-by-country-study/ Tue, 24 Apr 2018 11:48:35 -0000 Europe A strategic agreement has been signed by GlaxoSmithKline (GSK) and Orchard Therapeutics, involving the transfer of GSK’s portfolio of approved and investigational rare disease gene therapies to Orchard. https://www.epmmagazine.com/pharma-manufacturing-news/gsk-orchard-therapeutics-sign-strategic-agreement/ https://www.epmmagazine.com/pharma-manufacturing-news/gsk-orchard-therapeutics-sign-strategic-agreement/ Thu, 12 Apr 2018 13:06:21 -0000 Sign agreement This year will be big for Duchenne muscular dystrophy (DMD) as interest in treatments is on the rise and deal activity is increasing, reported data and analytics company, GlobalData. https://www.epmmagazine.com/pharmaceutical-industry-insights/2018-set-to-be-big-for-duchenne-muscular-dystrophy/ https://www.epmmagazine.com/pharmaceutical-industry-insights/2018-set-to-be-big-for-duchenne-muscular-dystrophy/ Wed, 21 Mar 2018 14:31:54 -0000 Year ahead 2018 Sanofi and RNAi therapeutics company, Alnylam Pharmaceuticals, have begun strategic restructuring of their RNAi therapeutics alliance to streamline and optimise development and commercialisation of certain products https://www.epmmagazine.com/pharma-manufacturing-news/sanofi-and-alnylam-are-restructuring-rnai-therapeutic-allian/ https://www.epmmagazine.com/pharma-manufacturing-news/sanofi-and-alnylam-are-restructuring-rnai-therapeutic-allian/ Mon, 08 Jan 2018 12:55:37 -0000 Restructuring A consultation by NICE and NHS England on changes to the arrangements for evaluating and funding medicines could potentially stop the flow of new medicines reaching patients with rare and complex diseases. https://www.epmmagazine.com/pharma-manufacturing-news/nice-and-nhs-england-consultation-could-stop-access-to-new-m/ https://www.epmmagazine.com/pharma-manufacturing-news/nice-and-nhs-england-consultation-could-stop-access-to-new-m/ Tue, 17 Jan 2017 12:15:00 -0000 Rare Diseases