The amount of cash inflow from collaborative agreement that was received as an upfront payment upon closing.

No definition available.

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- Definition

Amount of development milestone obligation revenue received during the period.

+ References

No definition available.

+ Details

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- Definition

Represents the percentage reduction in disease progression observed in treated participants compared with a specified comparator population.

+ References

No definition available.

+ Details

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- Definition

Represents the age of participants eligible for enrollment in a clinical trial.

+ References

No definition available.

+ Details

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- Definition

Represents the period, covered by participant data included in an interim analysis of a clinical trial.

+ References

No definition available.

+ Details

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- Definition

Represents the number of individuals expected to enroll in clinical trial.

+ References

No definition available.

+ Details

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Data Type:	xbrli:integerItemType
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- Definition

The number of products of the company.

+ References

No definition available.

+ Details

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Data Type:	xbrli:integerItemType
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- Definition

Ratio used to randomize participants between treatment and control groups in a clinical trial.

+ References

No definition available.

+ Details

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- Definition

Represents the period over which a clinical trial or study is conducted.

+ References

No definition available.

+ Details

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- Definition

Period of votoplam treatment evaluated in comparison with an external natural history cohort.

+ References

No definition available.

+ Details

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- Definition

Amount of investigational product administered to participants in a clinical trial.

+ References

No definition available.

+ Details

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- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

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- Definition

Amount of accumulated undistributed earnings (deficit).

+ References

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The Company (Details) $ in Thousands	1 Months Ended		9 Months Ended
The Company (Details) $ in Thousands	Apr. 30, 2026 USD ($) item age	Jan. 31, 2025 USD ($)	Sep. 30, 2026 USD ($) product	Dec. 31, 2025 USD ($)
Collaboration and royalty revenue
Number of products \| product			2
Accumulated deficit			$ (2,935,235)	$ (2,964,229)
PROVE-FA
Collaboration and royalty revenue
Number of individuals expected to enroll \| item	120
Study duration, clinical trial	84 days
PROVE-FA \| Minimum
Collaboration and royalty revenue
Eligible age, clinical trial \| age	7
PROVE-FA \| Maximum
Collaboration and royalty revenue
Eligible age, clinical trial \| age	21
Novartis
Collaboration and royalty revenue
Development milestone obligation revenue received			50,000
License and Collaboration Agreement \| Novartis
Collaboration and royalty revenue
Cash proceeds from upfront payment on collaboration agreement		$ 1,000,000
License and Collaboration Agreement \| Novartis \| Phase 3 Votoplam trial
Collaboration and royalty revenue
Number of individuals expected to enroll \| item	770
Randomization ratio, clinical trial	1.5
Treatment dosage, clinical trial (milligrams) \| item	10
Development milestone obligation revenue received	$ 50,000		$ 50,000
Interim analysis period, clinical trial	24 months
Treatment comparison period, clinical trial	24 months
Disease progression slowing percentage	52