Homocystinuria due to cystathionine beta-synthase deficiency

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At a Glance

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Regulatory Watchboard

1 event
Jun 2019

Thiola: FDA approved

THIOLA EC is indicated, in combination with high fluid intake, alkali, and diet modification, for the prevention of cystine stone formation in adults and pediatric patients 20 kg and greater with severe homozygous cystinuria, who are not responsive to these measures alone.

FDAcompleted

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

1

Total programs

1

Open now

1

Copay cards

Copay Assistance1

Betaine Anhydrous

Eton Pharmaceuticals, Inc.

OpenContact for details

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Approved Treatments

2 FDA-approved

Cystadane

(BETAINE)Orphan drugstandard

Recordati Rare Diseases

Methylating Agent [EPC]

FDA label ↗

Betaine Anhydrous

(BETAINE ANHYDROUS)Orphan drugstandard

Eton Pharmaceuticals, Inc.

Methylating Agent [EPC]

FDA label ↗

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Clinical Trial Landscape

No active clinical trials currently recruiting for Homocystinuria due to cystathionine beta-synthase deficiency.
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Specialist Network

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