Investigational therapy for SMA developed by PTC & H. La Roche.
A company that has the global licence for an investigational SMA therapy.
An approved administration route of SMA Gene therapy.
The Global Biogen campaign for SMA.
A group of drugs have been researched in many diseases one of them is SMA.
The First drug Approved for All SMA patient types.
Phase II trial that evaluate safety & tolerability of Nusinersen in pre-symptomatic newborns that are most likely to develop SMA based on their genetics.
The first four doses of SPINRAZA.
One of SMA disease Burdens that affects the QoL & increase the parental stress.
The company that has the marketing authorization for SMA gene therapy in MENA region.
A company that had been established in 1961, operating in MENA & Provide access to the best treatment options to healthcare professionals and to patients.
Down
Discounts or rebates in form of free provision of doses.
A promise of total value & services to be delivered & communicated to the customers.
One of SPINRAZA values that results in increased overall survival and increased incremental QALYs, which are associated with the achievement of motor milestones not normally observed in the natural history of SMA.
Pricing strategy tied to utilization of the drug.
Usage of two drugs successively for the same patient to treat certain disease.
The first & only gene therapy approved for SMA under 2 years.
A function within the company that work to pack the data to the right customer in the right time & cross functionality is one of its main pillars.
Governmental & Non-Governmental officials who are involved in commercial decisions related to drugs procurement.
a sum of money due as one of several equal payments for something, spread over an agreed period of time.
The route of SPINRAZA administration.
A company that has one of the largest rare diseases portfolio in MENA region & had contributed in changing SMA patients' lives in the region.
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