Rare Disease - Focus on the patient, follow the science
Sporadic inclusion body myositis (sIBM) is the most common acquired muscle disease of people over 50 and has no approved treatment. Finding therapies for rare disease flows from our desire to help patients underserved by today’s medicines.
What’s all this talk about rare disease?
The size of the patient population does not drive our research. Do we understand the science? Is there is unmet medical need? Those are the questions the Novartis Institutes for BioMedical Research asks. Surprising for a Pharma Organization? Not really. Here’s why.
Rare Diseases Beyond the Numbers
Novartis Institutes for BioMedical Research created this interactive quiz, hoping that testing people’s knowledge of rare diseases would help them understand the great need for new treatment options for patients with rare diseases.
Toward precision medicine for a rare blood cancer
Novartis has joined forces with lymphoma experts in academia to hack into the genetics of a rare form of blood cancer known as mantle cell lymphoma (MCL). Working with lymphoma experts from Charité – Universitätsmedizin in Berlin and the BC Cancer Research Centre in Vancouver, Canada, Novartis scientists provide evidence of two related but mutually exclusive molecular signaling pathways at play in MCL cell lines.
A rare disease, a challenging clinical trial
Cushing’s is a rare disease that affects only about 10 to 15 people per million. It is difficult to diagnose and can make clinical trials for such a small patient population very challenging. But finding treatments for patients with few options is at the heart of our approach. Learn more about one trial and the scientists and patients who made it work.
Letters To My Younger Self - Renee Bergeron
“I was always planning and very deliberate. I look back now and wish I hadn’t been so deliberate…” Renee Bergeron writes to herself at the age of 38, on her first day back in the office following the birth of her first daughter.