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orphan diseases
This page shows the latest orphan diseases news and features for those working in and with pharma, biotech and healthcare.
PMEA 2020: continuing to recognise excellence in improving patient outcomes
Excellence in Rare Diseases and Orphan Drugs categories. ... with support from Learner Adams Bones Ltd. 8. Excellence in Rare Diseases and Orphan Drugs.
Latest news
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Building a brighter future for rare disease patients
PMC is proud to work with several orphan diseases organisations with innovative technology platforms and support their medical affairs efforts. ... The calibre of the shortlist in the Excellence in Rare Diseases, and Orphan Drugs category at the PMEA
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Raising awareness and encouraging support on Rare Disease Day 2020
These include:. New requests for applications for the Orphan Products Grants programme, which supports natural history studies and clinical trials for rare diseases. ... Raising awareness is central to our mission to improve outcomes for people affected
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Biotech now vital to pipelines – but success rates falling
Among these drugs, 27% were treatments for cancer and its symptoms and 20% are for the treatment of infectious diseases. ... Almost half had orphan drug status and over a third were identified by the FDA as first-in class therapies – such as
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Poseida gets Novartis backing for myleoma CAR-T challenger
in single-treatment cures for numerous oncologic indications and orphan genetic diseases, with an initial focus on chimeric antigen receptor T cell (CAR-T) therapies.”.
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‘Inflexible’ NICE blocking access to rare disease drugs
patients. However many rare diseases do not qualify for this pathway, and must therefore be appraised via its standard Single Technology Appraisal (STA) process. ... UK. “This system will continue to fail people living with rare diseases until the
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Latest Intelligence
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Listen, learn and lift off
The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) are expecting a busy year for approvals, with frantic activity in orphan diseases as gene and cell therapy ... That has completely changed and we’re now launching
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Piloting a route to the biosimilars opportunities in Europe
Biological medicines represent a major advance in the treatment of serious pathological conditions such as cancer, neurodegenerative and autoimmune diseases, in the form of biosimilars. ... For orphan designations (medicine for rare diseases),
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Assessing the impact of disease prevalence on access and pricing of rare disease drugs in England
Comparison of orphan drug assessments from NICE versus the NHS. The question of how to adequately capture the holistic value of orphan drugs (ODs) for rare diseases is the subject of ... to drugs targeting ‘very rare’ diseases (typically ultra-orphan
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Biopharma and orphan drugs
Rare disease regulations. Twenty one of the 48 novel drugs approved in the US were for rare or orphan diseases – those that affect fewer than 200, 000 citizens. ... Clinical complications. Given the rarity of orphan diseases, one of the greatest
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Industry needs an aligned position on the future of the EU OMP Regulation
The Pharmaceutical Strategy is likely to include innovation-boosting measures and plans to revise the EU Regulation on Orphan Medicinal Products (OMP Regulation). ... of orphan diseases).
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Latest appointments
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Amryt Pharma appoints Jordi Casals as head of Europe
He joins the company from Aegerion Pharmaceuticals. Amryt Pharma, a company focused on treatments for rare and orphan diseases, has appointed Jordi Casals as the biopharma’s head of Europe.
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Sobi appoints Norbert Oppitz SVP, speciality care
He joins the rare disease pharma firm from BSN Medical. Rare diseases healthcare company Swedish Orphan Biovitrum (Sobi) has appointed Norbert Oppitz as its senior vice president for its newly established
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Minoryx Therapeutics adds to board of directors
He brings over 30 years of regulatory strategy expertise to the orphan diseases specialist, and currently also serves as a board member at Sarepta Therapeutics.
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Minoyrx Therapeutics appoints chief medical officer
Dr Uwe Meya joins from Lupin Atlantis Holdings. Spanish orphan disease specialist Minoryx Therapeutics has appointed Dr Uwe Meya as its new chief medical officer. ... He will also support the further development of our pipeline in orphan diseases.”.
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Ovid Therapeutics adds member to scientific advisory board
Ovid is a privately-held biopharmaceutical company focused on developing therapies for rare and orphan diseases of the brain. ... He said: “Ovid is pursuing an ambitious approach of tackling rare neurological diseases with significant unmet needs.
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Latest from PMHub
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Onyx Health Joins Forces with Rare Disease Specialists Actigen
Actigen's work focuses on identifying and developing biological medicines for rare diseases with unmet medical needs. ... At Actigen, our mission is to improve the lives of individuals with rare diseases, and we hope that GNR-055 will be the start of
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How to Simplify Genetics & Raise Awareness Around Orphan Diseases
Gail Ouellette, President and Scientific Director of Regroupement Québécois des Maladies Orphelines ( RQMO)/Quebec Coalition of Orphan Diseases and Creator of the “ Genetics Simply" website, shares her thoughts on the barriers ... faced by rare
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Lucid Group has four programmes shortlisted for PMEA 2019
Category: Excellence in Innovation. DermDialogue: Bringing innovation to MSL‐led education. Excellence in Rare Diseases and Orphan Drugs.
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Random42 attending Biotech Showcase in San Francisco
This year’s agenda includes a focus on six therapeutic areas that are seeing intriguing research, innovative technologies, and the promise of transforming the way diseases are treated across the globe:. ... Alzheimer’s Disease. Cell and Gene Therapy.
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Another spellbinding result for Lucid Group at the 2018 PMEAs
The first award was gained in the category, ‘Excellence in Rare Diseases and Orphan Drugs’for, Revolutionising Management of Hidradenitis Suppurativa in Europe: Why?
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Approximately 2 fully matching, plus 19 partially matching documents found.
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