rare diseases
Rare diseases – spinal muscular atrophy
January 30, 2026Medical Communications gene therapy, medical communications, muscle and motor function, muscle weakness, rare diseases, sma
Spinal muscular atrophy (SMA) is a rare disease characterised by voluntary muscle weakness that can lead to difficulties in activities …
Biogen and Stoke report positive results for Dravet syndrome drug
July 17, 2025Research and Development Biogen, Dravet Syndrome, Neurology, Stoke Therapeutics, epilepsy, rare diseases
Biogen and Stoke Therapeutics have shared encouraging new data for their experimental treatment, zorevunersen, which is being developed for Dravet …
MHRA approve new treatment combo for transplant-ineligible multiple myeloma patients
January 30, 2025Research and Development MHRA, Oncology, approvals, multiple myeloma, rare diseases
The Medicine and Healthcare products Regulatory Agency (MHRA) has approved quadruplet therapy, SARCLISA (isatuximab) in combination to treat multiple myeloma …
Results from Novadip phase 1b/2a trial reveal 88% efficacy
January 28, 2025Clinical Research Congenital Pseudoarthritis of the Tibia, Rare Diseases, clinical trials, efficacy, paediatric, rare diseases
Novadip Biosciences, a clinical stage biotechnology company with a specialty in regenerative medicine, has announced the results of its 12-month …
Chiesi announces phase 3 results from enzyme-replacement study
October 29, 2024Research and Development Internal Medicine, Rare Diseases, chiesi Global rare diseases, clinical trials, pharma, rare diseases
Chiesi Global Rare Diseases have announced the results from their BRIGHT trial, which evaluates the efficacy of enzyme-replacement therapy drug …
AstraZeneca to acquire Amolyt Pharma for up to $1.05bn
March 14, 2024Business Services Amolyt Pharma, AstraZeneca, Rare Diseases, acquisition, rare diseases
AstraZeneca has announced that it has entered into a definitive agreement under which it will acquire Amolyt Pharma for up …
Saniona appoints Rami Levin as President and Chief Executive Officer
January 8, 2020Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing appointments, rare diseases
Saniona has announced that Rami Levin will take over as the Chief Executive Officer of the company as it seeks …
Roche scores two EU approvals in haemophilia and rare autoimmune disease
March 15, 2019Medical Communications EC, EU, Europe, Roche, haemophilia, rare diseases
The European Commission (EC) has approved Roche’s haemophilia treatment Hemlibra (emicizumab). The approval comes as the EC approved Roche’s MabThera …
China to establish national rare disease network
February 19, 2019Manufacturing and Production China, NHC, healthcare, rare diseases
China is set to establish a national collaborative network of hospitals for rare disease diagnosis and treatment to promote early …
The Autumn 2018 edition of Pharmafile is available to read online now!
December 18, 2018Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing immunology, infectious diseases, pharma, pharmafile, rare diseases
The Autumn 2018 edition of Pharmafile, our long-running bi-annual guide to the latest trends, projections and thought leadership positions in …
FDA approves AstraZeneca’s Lumoxiti for hairy cell leukaemia (HCL)
September 17, 2018Sales and Marketing AstraZeneca, HCL, Lumoxiti, leukaemia, rare diseases
AstraZeneca’s Lumoxiti has been approved for use for the treatment of relapsed or refractory hairy cell leukemia (HCL)in adults who …
Global orphan and rare dermatology market set to exceed $6 billion by 2024
August 31, 2018Medical Communications Orphan Drugs, analysis, dermatology, market reports, rare diseases
The global orphan and rare dermatology premium products market is expected to grow rapidly in the next half a decade …
FDA approves RNA therapy for rare genetic disease
August 13, 2018Sales and Marketing FDA, Onpattro, Orphan Drugs, RNA, rare diseases
The US Food and Drug Administration has approved the RNA-based therapy Onpattro, for the treatment of the rare genetic disease …
The need for flexible appraisal of rare disease medicines
March 15, 2018Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Amryt Pharma, feature, pharma, rare disease, rare diseases
Recent data has shown that England is, on average, slower than Germany, France, Spain and Italy in making orphan medicinal …
Taking science and making people feel connected to it
August 21, 2017Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Pfizer, biotech, drugs, pharma, pharmaceutical, rare diseases
Owen Marks, Country Head for the Rare Disease Unit at Pfizer UK, discusses his career so far and what drives …
