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StemRIM (4599) · FactDownward revision to estimated patient numbers

The rarity of recessive dystrophic epidermolysis bullosa and unmet treatment needs

First written in FY2022-Q3. 6 of 9 entries say "Restated". Latest entry: FY2026.

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ProgressHeldSoftenedWorseRestatedFY2022-Q3 – FY20262022: 320222023: 120232024: 220242025: 120252026: 22026
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The rarity of dystrophic epidermolysis bullosa and constraints on treatment developmentA theme 1 companies write about · 0 as a headwind · 0 as a tailwind

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  1. FY2026Downward revision to estimated patient numbersAnnual report

    「The target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease with an estimated nationwide patient population of approximately 300. Planning a large-scale Phase III trial is difficult, and currently available effective treatments are limited.」

    The estimated nationwide patient population for dystrophic epidermolysis bullosa was revised from approximately 400 to approximately 300. The explanation that effective treatments are limited and that a large-scale Phase III trial would be difficult remains unchanged.
  2. 2026-06-18RestatedOccurring Fact

    「There is currently no curative treatment for dystrophic epidermolysis bullosa, and developing one is an urgent challenge in modern medicine.」

    The absence of a curative treatment for dystrophic epidermolysis bullosa and the need for a new treatment were reconfirmed.
  3. FY2025RestatedAnnual report

    「For the epidermolysis bullosa treatment, the target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease estimated to affect approximately 400 people nationwide, for which no effective treatment currently exists. It is difficult to plan a large-scale Phase III clinical trial.」

    The patient population for dystrophic epidermolysis bullosa, the absence of an effective treatment, and the difficulty of conducting a large-scale Phase III clinical trial have been reaffirmed.
  4. FY2024RestatedAnnual report

    「For the epidermolysis bullosa treatment, the target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease estimated to affect approximately 400 people nationwide, for which no effective treatment currently exists. It is difficult to plan a large-scale Phase III clinical trial.」

    The patient population of approximately 400 for dystrophic epidermolysis bullosa, the absence of an effective treatment, and the difficulty of conducting a large-scale Phase III clinical trial have been reaffirmed.
  5. FY2024-Q3RestatedAnnual report

    「For the epidermolysis bullosa treatment, the target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease estimated to affect approximately 400 people nationwide, for which no effective treatment currently exists. It is difficult to plan a large-scale Phase III clinical trial.」

    The patient population of approximately 400 for dystrophic epidermolysis bullosa, the absence of an effective treatment, and the difficulty of conducting a large-scale Phase III clinical trial have been reaffirmed.
  6. FY2024-Q1RestatedAnnual report

    「For the epidermolysis bullosa treatment, the target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease estimated to affect approximately 400 people nationwide, for which no effective treatment currently exists. It is difficult to plan a large-scale Phase III clinical trial.」

    The patient population of approximately 400 for dystrophic epidermolysis bullosa, the absence of an effective treatment, and the difficulty of conducting a large-scale Phase III clinical trial have been reaffirmed.
  7. FY2023-Q1RestatedAnnual report

    「For the epidermolysis bullosa treatment, the target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease estimated to affect approximately 400 people nationwide, for which no effective treatment currently exists. It is difficult to plan a large-scale Phase III clinical trial.」

    The patient population of approximately 400 for dystrophic epidermolysis bullosa, the absence of an effective treatment, and the difficulty of conducting a large-scale Phase III clinical trial have been reaffirmed.
  8. FY2022Change in the estimated patient populationAnnual report

    「For the epidermolysis bullosa treatment, the target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease estimated to affect approximately 400 people nationwide, for which no effective treatment currently exists. It is difficult to plan a large-scale Phase III clinical trial.」

    The nationwide estimate for dystrophic epidermolysis bullosa patients has changed from approximately 200 to approximately 400. The explanation that the disease is rare, has no effective treatment, and makes a large-scale Phase III clinical trial difficult remains unchanged.
  9. FY2022-Q3First statedAnnual report

    「The target disease, dystrophic epidermolysis bullosa, is a rare and intractable disease affecting approximately 200 people nationwide, for which no effective treatment currently exists. The number of new patients each year is estimated at approximately 15, making it difficult to plan a large-scale Phase III clinical trial.」

    It was stated that dystrophic epidermolysis bullosa affects approximately 200 people nationwide, with approximately 15 new patients each year, that no effective treatment currently exists, and that planning a large-scale Phase III clinical trial is difficult.
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