Oft genetisch bedingt und medizinisch komplex. Unser Beitrag zeigt Ihnen alles Wichtige! Welche seltenen Erkrankungen gibt es? Erhalten Sie Definition, Zahlen und Beispiele.
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Vor einem Tag · The UK's National Health Service (NHS) has introduced Pfizer's tafamidis for transthyretin amyloidosis cardiomyopathy (ATTR-CM), a rare heart condition characterised by shortness of breath ...
Vor 2 Tagen · hPS cells have primarily been used to study rare diseases 6,7, most of which have been associated with a few genetic loci, typically located in protein-coding genome regions. hPS cells have been ...
Vor einem Tag · Esteve to acquire Perrigo’s HRA pharma rare diseases business. 29-04-2024. Family-controlled Spanish pharma company Esteve has signed a binding offer with USA-based Perrigo Company to acquire the latter’s HRA Pharma Rare Diseases, a specialist in rare and ultra-rare diseases.
Vor 2 Tagen · Pfizer’s sickle cell disease med Oxbryta earned a key thumbs up from England’s National Institute for Health and Care Excellence (NICE), allowing some 4,000 patients to benefit from the treatment.
Vor 5 Tagen · There are several ways to do this: replacing the mutated gene that has caused the disease with a healthy copy of the gene; ‘switching off’ a mutated gene that is functioning incorrectly or adding...
Vor 3 Tagen · Sickle Cell Disease (SCD) is a significant public health concern in the GCC countries, with prevalence rates ranging from 0.24% to 5.8%; To further highlight its commitment on improving patient outcomes in the Gulf, Pfizer hosted a media roundtable to raise awareness on Sickle Cell Disease (SCD) and revealed key findings from Sickle Cell Health Awareness, Perspectives, and Experiences (SHAPE ...
Vor 5 Tagen · National Organization for Rare Disorders (NORD) Names Pamela K. Gavin as CEO; Peter L. Saltonstall Retires and Will Serve in Consultative Role. QUINCY, MA, May 1, 2024—The National Organization for Rare Disorders (NORD) announced today that Pamela K. Gavin has been named CEO effective immediately. She succeeds Peter L. Saltonstall who ...
