Application for Treatment

There is a substantial amount of information concerning the patient that needs to be submitted with the application in order for n-Lorem and the Access to Treatment Committee to make decisions concerning the potential to treat each patient with an experimental ASO.

Included in the submission are:

• Diagnosis
• Characterization of the mutation
• Functionalization of the gene
• Clinical pathophenotype
• Organs affected
• Primary manifestations of the mutation to be treated
• Other disease manifestations
• Goals of the treatment (e.g. what symptoms do you expect to improve)

Read more about our criteria and
qualifications:

Qualifications for Treatment

Strengths and limitations of ASO technology:

ASO technology is validated, versatile (multiple post-RNA binding mechanisms), effective in many organs at low doses, deliverable by many routes of administration, and active both systemically and locally. Moreover, ASO medicines of each specific chemical class behave similarly, facilitating dose and schedule selection. However, as is the case for all drug discovery technologies, ASO technology has limits. ASO technology cannot replace genes, therefore, true null mutations may be better treated with other therapeutic approaches. Compound heterozygous mutations are unlikely to be treatable by ASOs, or if the gene in question serves a vital function, then reducing the transcript from that gene may be too risky.