Amicus Therapeutics ((FOLD)) announced an update on their ongoing clinical study.
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Amicus Therapeutics is conducting a study titled ‘Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001),’ aimed at providing treatment access to migalastat hydrochloride for individual patients with Fabry disease. The study’s key objective is to offer treatment to patients with specific genetic mutations who lack access to commercial Galafold or other treatment alternatives, highlighting its significance in addressing unmet medical needs.
The intervention being tested is migalastat hydrochloride, available in 150 mg capsules and 20 mg dispersible tablets, intended to treat Fabry disease by targeting specific genetic mutations.
The study follows an expanded access design, allowing physicians to request treatment for eligible patients. It is open-label, meaning both doctors and patients know the treatment being administered, with the primary purpose of providing treatment access.
The study began on November 17, 2011, with the latest update submitted on July 1, 2025. These dates are crucial as they reflect the study’s long-term commitment to patient access and ongoing updates to ensure relevance and accuracy.
This update could positively influence Amicus Therapeutics’ stock performance by reinforcing its commitment to patient care and innovation. It may also impact investor sentiment positively, especially in the context of limited competition in this niche market.
The study is ongoing, with further details available on the ClinicalTrials portal.