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rare disease
This page shows the latest rare disease news and features for those working in and with pharma, biotech and healthcare.
FDA priority review for Roche’s Esbriet in unclassifiable interstitial lung disease
Interstitial lung disease (ILD) encompasses a diverse group of over 200 types of rare pulmonary diseases. ... We are working closely with the FDA in the hopes of offering Esbriet to people with UILD, a rare and debilitating disease,” he added.
Latest news
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Pfizer makes first investments through ‘breakthrough growth’ fund
rare disease, vaccine and hospital. ... The first $120m was invested during the second half of 2020, Pfizer announced in a statement, and went to four biotechs developing therapies ranging from immunotherapies to rare genetic disease.
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Novartis receives FDA breakthrough therapy designation for rare disease med
Swiss pharma company Novartis has received a breakthrough therapy designation (BTD) for its experimental medication iptacopan for the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH). ... The therapy, also known as LNP023, was also awarded a
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Lilly picks up gene therapy programme in $1bn Prevail Therapeutics acquisition deal
Prevail’s pipeline spans clinical-stage and preclinical neuroscience assets, including lead gene therapies PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) ... It has also been granted
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AstraZeneca to acquire Alexion for $39bn
By EvaluatePharma’s forecasts, the global rare disease market is set to grow by a low double digit percentage until 2026. ... We bring to AstraZeneca a strong portfolio, an innovative rare disease pipeline, a talented global workforce and strong
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PMEA 2020: continuing to recognise excellence in improving patient outcomes
with support from Learner Adams Bones Ltd. 8. Excellence in Rare Diseases and Orphan Drugs. ... Winner: I Am Number 17 – A Rare Disease Awareness Campaign by Takeda UK with support from Hill Knowlton Strategies.
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Latest Intelligence
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Sourcing R&D innovation: why pharma companies need to evolve their business models
These higher prices are often supported by the fact that many rare disease populations have limited or no approved treatments available with an acceptable efficacy and safety profile. ... Companies developing rare disease drugs can also often secure
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Successful product commercialisation: what can we learn from the rare disease setting?
Establishing a successful launch strategy. While each rare disease launch is different, several similarities are shared – a key element being a truly patient-centric, cross-functional approach. ... So, can lessons be learned from the rare disease
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Not waving but drowning – reflections on data curation in the age of information overload
The growth in rare disease congresses is testament to this. All these meetings need content, that is, data to be shared as abstracts, presentations and posters that the authors hope will
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COVID-19 – a catalyst for technology adoption in clinical trials
Finally, Raremark, another AlbionVC portfolio company with a novel patient engagement technology enables the finding and remote engagement of rare disease patients. ... These patients are hard to reach in normal times and so the company is reporting
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The impact of COVID-19 on clinical trials
Trial size – trials with minimal margins for determining statistical significance and trials evaluating rare disease drugs (eg, where patient accrual is already difficult and the pandemic increases the risk of patient ... These impacts could be
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Approximately 2 fully matching, plus 106 partially matching documents found.
Latest appointments
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Syneos Health appoints trio of senior hires
including Omnicom, regional and global roles, and has deep therapeutic expertise in oncology and rare disease.
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Azafaros expands executive team with three senior hires
While at Sanofi, she served as head of rare diseases, Central and Eastern Europe – in this role, she led all commercial activities for rare disease-related programmes in those regions. ... The insights and experience provided by Kyle, Ruben and Louise
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New hires at Crescendo, Healx and Galecto
He co-founded the rare disease patient group support charity Findacure with Nick Sireau, the advisor to and the inspiration behind Healx. ... Hall’s experience in drug development includes significant contributions to the repurposing of nitisinone for
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Health comms agency emotive hires new cell and gene therapy leader
The agency says a large proportion of its work is now in the field of rare and ultra-rare disorders, where advanced therapies will play an important part in treatment protocols. ... product in a rare and previously untreatable eye disease.
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Ipsen hires general manager, Laura Hamill joins Gilead and Orchard expands leadership team
expertise as we shape our business for future growth with an unrelenting commitment to enhancing the lives of patients living with rare disease.
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Latest from PMHub
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Real-world evidence: breaking boundaries in rare disease
However, small patient populations, the high unmet need, and difficulties identifying a control group make it difficult to conduct an RCT when trialling a rare disease therapy. ... Sonal Bhatia, Vice President, Medical Affairs Rare Disease at Pfizer,
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Unpacking rare diseases in the first edition of Delta magazine
Fishawack Health launches Delta magazine with a deep dive into rare disease commercialization. ... Navigating real-world evidence and why it could be a gamechanger in rare disease.
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emotive supports Rare Disease Day 2019
With a series of events and activities over the week leading up to Rare Disease Day on February 28th, we showed our support for stimulating greater awareness of Rare Diseases along ... In line with the Rare Disease Day theme, we took photos of our team
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Porphyria Awareness Week – Round Up
Porphyria Awareness Week has given the team at emotive an opportunity to show our support for those touched by this debilitating Rare Disease as well as our enthusiasm for working in ... Porphyria is a rare condition that is thought to affect around 1 in
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Learning about Rare Genetic Disorders on Undiagnosed Children’s Day
Children can be left facing an uncertain future and an unclear treatment pathway when they have a rare disorder. ... A Rare Disease UK report published in 2016 found that nearly half of the respondents who identified as undiagnosed had been waiting
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Approximately 13 fully matching, plus 86 partially matching documents found.
COVID-19 Updates and Daily News
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- Nucleus Global announces a trio of promotions
- Moderna cooperating with investigation into possible COVID-19 vaccine allergic reactions
- Sinovac’s COVID-19 vaccine appears safe in older adults
- Valneva set to begin commercial manufacturing of COVID-19 vaccine within days
- Pfizer, BioNTech temporarily slow down shipments of COVID-19 vaccine to EU
- Lilly’s antibody bamlanivimab cuts COVID-19 risk by up to 80%
- Mistrust in medical research: a patient perspective
- Real-world evidence: breaking boundaries in rare disease
- Live Webinar: Importance of Highlighting Patient Voices, Workforce Well-Being, and Health Literacy During and After the Pandemic
- The heavy toll of COVID-19 on cancer patients
- Unpacking rare diseases in the first edition of Delta magazine
- Virtual HealthTech conference powered by Impetus Digital: A Customer Story
PMHub
11 London
11 London is a strategic and creative agency working in the fields of health and humanity – complex areas, but...
Latest intelligence
- Mistrust in medical research: a patient perspective
- The recent development of several COVID-19 vaccines has placed medical research firmly in the spotlight, highlighting public confusion and misinformation about clinical trials. Patient advocate, Trishna Bharadia reveals what the...
- Real-world evidence: breaking boundaries in rare disease
- Generating data for drug launches is a challenging process. In rare diseases, with small patient populations and high unmet need, evidence generation is even more complex. Consultant Sarah Poole and...
- The heavy toll of COVID-19 on cancer patients
- We all know that finding and treating cancer early saves lives. During the COVID-19 pandemic, oncology treatments paused while cancer continued to spread. So, what has been the impact of...
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