Munich news, health insurance, technology, jobs and other topics for expatriates. The Eye Newspapers covers daily news and offers services for foreigners.
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...
We need a different way to pay for these “miracle” drugs. One option is to have them paid for on an annual basis, spreading the cost over the patient’s estimated lifespan, with payment to stop if the ...
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
An intravenous form of the gene therapy is already approved and sold as Zolgensma in the US for patients less than 2 years of age.
The FDA wants to end bulk production of copycat versions of weight loss drugs; telehealth companies and compounding ...
After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.
Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular ...
Novartis released two Phase 3 datasets on Wednesday that show a new version of its gene therapy Zolgensma led to motor ...
Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...
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