rare disease
Celgene’s Inrebic becomes second FDA-approved therapy for rare bone marrow disorder
August 19, 2019Sales and Marketing Cancer, Cerlgene, FDA, Inrebic, pharma, rare disease
Celgene is celebrating the approval in the US of its Janus kinase (JAK) 2 inhibitor Inrebic (fedratinib) in the treatment …
Regeneron therapy halves LDL cholesterol levels in rare genetic condition
August 15, 2019Research and Development Regeneron, evinacumab, pharma, rare disease
Regeneron has reported a strong showing for its investigational angiopoietin-like 3 (ANGPTL3) antibody evinacumab in the treatment of the rare …
Living with a rare genetic neurodevelopmental disorder
August 5, 2019Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing KAT6A, feature, patient experience, pharma, rare disease
Vaila Morrison’s daughter Eilidh was born with the ultra-rare condition KAT6A. As a designer and architect by profession, Vaila told …
Living with mal de debarquement syndrome
July 1, 2019Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing patient experience, pharma, rare disease
Polly Moyer reflects on her experiences living with the rare condition mal de debarquement syndrome, and discusses how connecting with …
Alnylam and Sanofi close research phase of 2014’s $700m RNAi rare disease partnership
April 8, 2019Research and Development, Sales and Marketing Alnylam, Genzyme, Sanofi, pharma, rare disease
Alnylam and Sanofi have announced their intention to bring to an end the research and option phase of a $700 …
Patient Experience: Living with two rare diseases
March 18, 2019Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Wyburn-Mason Syndrome, acromegaly, feature, patient experience, pharma, rare disease
Dan Jeffries was diagnosed with the ultra-rare condition Wyburn-Mason syndrome at a young age, leaving him blind in one eye. …
Akcea and Ionis’ Waylivra nets CHMP approval in ultra-rare familial chylomicronaemia syndrome
March 5, 2019Manufacturing and Production, Sales and Marketing Akcea, FCS, Ionis, familial chylomicronemia syndrome, pharma, rare disease
Akcea Therapeutics and Ionis Pharmaceuticals have announced that their investigational therapy Waylivra (volanesorsen) has been recommended by the EMA’s Committee …
Read the March 2019 issue of Pharmafocus online now!
February 26, 2019Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Cancer, Drug pricing, HIV, Trump, WHO, brexit, depression, mental health, pharmacovigilance, rare disease
Just one month to go until Brexit here in the UK, and it’s still all to play for. After months …
Ipsen shells out $1.31bn to acquire rare disease specialist Clementia Pharmaceuticals
February 25, 2019Sales and Marketing Clementia pharmaceuticals, acquisition, ipsen, rare disease
In a bid to boost its rare disease portfolio prospects, Ipsen has revealed its intention to acquire Clementia Pharmaceuticals, with …
Top Ten most popular articles on Pharmafile.com this week
February 22, 2019Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing AstraZeneca, Cancer, China, MedImmune, Xeljanz, asthma, diabetes, keytruda, osteoarthritis, pharma, rare disease
Happy Friday! With the weekend within reach, check out our run-down of the top ten most popular articles on Pharmafile.com …
Living with Alström syndrome
January 14, 2019Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Alström syndrome, patient experience, pharma, rare disease
Kay Parkinson lived the tragedy of seeing both of her children born with an ultra-rare condition, and the elusiveness of …
Promising interim Phase 3 data spurs epidermolysis bullosa trial expansion for Amryt
January 7, 2019Research and Development, Sales and Marketing Amryt, epidermolysis bullosa, pharma, rare disease
Rare disease firm Amryt has announced interim efficacy data for its epidermolysis bullosa (EB) therapy AP101, showing that things are …
Living with fibular hemimelia
November 8, 2018Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing cambridge rare disease network, diagnosis, fibular hemimelia, rare disease, therapy
Helena Baker, Vice President of Clinical Strategy at Medical Research Network and a trustee of the newly formed Rare Disease …
Alexion’s Soliris almost entirely eliminates relapses in rare neuromyelitis optica spectrum disorder
September 26, 2018Research and Development Alexion, neuromyelitis optica spectrum disorder, pharma, rare disease, soliris
Alexion has revealed new Phase 3 findings for Soliris (eculizumab), showing the drug met its primary endpoint in the treatment …
Ionis/Akcea’s ultra-rare disease drug rejected by FDA
August 28, 2018Manufacturing and Production, Sales and Marketing Akcea, FDA, Ionis, familial chylomicronemia syndrome, pharma, rare disease
The FDA has opted to refuse approval to Akcea and Ionis’ Waylivra (volanesorsen) for the treatment of the ultra-rare hereditary …
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