Please login to the form below
rare disease unit
This page shows the latest rare disease unit news and features for those working in and with pharma, biotech and healthcare.
UK’s Competition and Markets Authority to review AZ/Alexion merger
The Anglo-Swedish pharma company hopes to combine its own precision medicine expertise with Alexion’s specialist rare disease knowledge to leverage its capabilities in a number of therapy areas, including ... As part of these plans, AZ will make
Latest news
-
AstraZeneca reports ‘strong’ Q1 results, forecasts accelerated growth in 2021
AZ has also excluded its proposed acquisition of Alexion Pharmaceuticals from this guidance, adding in the Q1 results that Alexion will become its rare disease unit and an ‘area of expertise’.
-
Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial
Two patients had at least one year of follow-up and showed modest increases in the NorthStar Ambulatory Assessment (NSAA) scale, used to assess symptoms caused by the muscle-wasting disease. ... Pfizer adopted a cautious tone in its assessment of the data
-
Europe responding to gene therapy challenge, but picture remains fragmented
Last year saw the launch of three groundbreaking products in Europe: the CAR-T competitors, Kymriah from Novartis and Gilead’s Yescarta and Spark’s rare eye disease gene therapy Luxturna. ... Giorgio Iotti, head of programme leadership and control,
-
Sanofi exits R&D pact with Alnylam on rare disease
Exit also frees up $700m equity investment. Sanofi is bowing out of a five-year-old R&D alliance with Alnylam on rare disease therapies, but the partners will continue to ... Sanofi and Alnylam started working together on gene-silencing therapies in 2014,
-
Pfizer hedges bets on gene therapy deal
Pfizer's rare disease leader Seng Cheng. “VTX-801 could provide a potentially transformative therapeutic option for patients with Wilson disease by directly addressing the underlying cause of the disease—the ... inability to excrete copper owing to a
AstraZeneca reports ‘strong’ Q1 results, forecasts accelerated growth in 2021
Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial
Europe responding to gene therapy challenge, but picture remains fragmented
Sanofi exits R&D pact with Alnylam on rare disease
More from news
Approximately 1 fully matching, plus 31 partially matching documents found.
Latest appointments
-
AZ appoints Alexion’s Aradhana Sarin as chief financial officer
Dunoyer will then go on to become chief executive officer of the Alexion unit and chief strategy officer of AZ. ... I’m looking forward to working with him in his new role as CEO, Alexion, the Rare Disease Unit of AstraZeneca, and to his continued
AZ appoints Alexion’s Aradhana Sarin as chief financial officer
More from appointments
Approximately 0 fully matching, plus 1 partially matching documents found.
Latest from PMHub
-
Focus on Rare Diseases
Alison Cantle, Rare Disease Business Unit Director at Lucid Group, explains what this means for medical education agencies: “As pharmaceutical organisations focus on personalised medicine, the need for rare disease expertise ... Within this unit, Lucid
More from PMHub
Approximately 0 fully matching, plus 1 partially matching documents found.
COVID-19 Updates and Daily News
Featured jobs
- Senior Account Director & Associate Director - Healthcare Ad...
-
Highly Competitive Salary, Bonus &Benefits
-
Planner
-
Salary TBC
- FREELANCE Medical Writer, Senior Medical Writer, Principal Medic...
-
Highly Competitive Day Rate, Bonus &Benefits
- Medical Writer, Senior Medical Writer, Principal Medical Writer,...
-
Highly Competitive Salary, Bonus &Benefits
- Client Services Director – London or Home Based – Medical Commun...
-
Highly Competitive Salary, Bonus &Benefits
-
Publications Director
-
Competitive Salary
-
Senior Account Manager
-
Salary TBC
- Client Services Director, Patient Engagement & Solutions, Lo...
-
Highly competitive salary
- Senior Leadership Role – Medical Communications – North West
-
Highly Competitive Salary, Bonus &Benefits
- Editor & Senior Editor - Medical Communications - Home Based
-
Highly Competitive Salary, Bonus &Benefits
- Home Working Medical Copy Writer & Senior Medical Copy Write...
-
Highly Competitive Salary, Bonus &Benefits
- Group Account Director, Patient Engagement & Solutions, Lond...
-
Highly competitive salary
- Novavax announces further delays for regulatory filings of COVID-19 vaccine
- Novartis highlights potential of its radioligand therapy in prostate cancer at ASCO
- Merck signs $1.2bn supply deal with US government for Molnupiravir
- AZ, Pfizer/BioNTech COVID-19 vaccines 'highly effective' against Indian variant
- Piloting a route to the biosimilars opportunities in Europe
- FDA approves Biogen’s aducanumab for Alzheimer’s disease in ‘landmark’ decision
- Novartis’ iptacopan scores positive results in rare blood disorder PNH
- Sanofi’s C1s inhibitor sutimlimab hits the mark in late-stage CAD study
- bluebird bio presents new data for beti-cel in beta thalassemia at EHA2021
- Roche reveals four-year follow-up data for Venclyxto in CLL at EHA2021
- BeiGene’s reveals positive data for Brukinsa in head-to-head Imbruvica trial
- Building the hospital of the future
- First NHS patient treated with Novartis’ SMA gene therapy Zolgensma
- FDA approves Biogen’s aducanumab for Alzheimer’s disease in ‘landmark’ decision
- Sinovac’s COVID-19 vaccine appears safe in older adults
- Novo Nordisk says Victoza outperforms SGLT2 inhibitors
- FDA advisory committee overwhelmingly votes no on Biogen's aducanumab
- Pfizer CEO says oral COVID-19 pill could be ready by the end of the year
PMHub
Videum Health
Videum Health is a premium video platform that offers brands innovative engagement strategies to reach targeted healthcare audiences on a...
Latest intelligence
- Building the hospital of the future
- Senior Consultant Leah Carlisle explores the growing trend of virtual hospitals and what it takes to develop a holistic digital ecosystem centered on the ultimate patient experience....
- Why people join a clinical trial
- To understand why people decide to take part in clinical trials, you first need to understand how people make decisions....
- Article:
- The disparity of patient experiences with Crohn’s Disease in Europe and Japan...
