DEDICATION

Dedication is the driver our success.

Because at TRPharm no challenge is too big or too small. Whether it is finding solutions to seemingly impossible market access situations, generating new clinical data or creating marketing best practices that our partners want to propogate on a global level; once we have taken on the challenge, we will go all the way.

DEDICATED TO OVERCOMING CHALLENGES

A CASE EXAMPLE

Situation:

Low rate of diagnosis due to high screening cost

  • CTX is a rare metabolical disease see in 5-20 in a million newborns. Early diagnosis is critical to treatment outcomes

  • Although diagnostic tests are available, effective newborn screening strategies were infeasible due to rarity of the disease

  • 80-85% of CTX patients are expected to undergo juvenile cataract surgery. However, there was no data on the prevalence of CTX as an underlying cause of juvenile cataracts

  • Investigation of this link could create a cost effective screening solution and a paradigm shift in CTX

ACTIONS:

Retrospective screening of juvenile cataract surgeries

  • With prominent physicians in the field, we supported an observational, epidemiological study

  • 17 centers including state, public and university hospitals enrolled in the study

  • 279 patients were run with elimination through “mignari suspicion index”, “cholestanol test” and “genetic test”

You can download the related
scientific literature here
RESULTS:

A promising global solution for the diagnosis of CTX

  • 4 of those 279 patients were diagnosed as CTX, confirming the hypothesis of a 1-2% diagnosis rate in this small patient group

  • The study results were published at an international congress as the first of its kind

  • This establishes feasibility for a viable screening method that can reach most of the patients

Built on Transparency

Guided by Expertise

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