HealthQuill Rare Disease
The US Food and Drug Administration cleared PTC Therapeutics’ sepiapterin for treating children and adults with a rare genetic condition where the body cannot break down a part of protein called phenylalanine, which comes from foods like meat, eggs, and milk.
Drugs Health Pharma

US regulator clears PTC’s medicine to treat a rare genetic metabolic disease 

HQ Team July 29, 2025: The US Food and Drug Administration cleared PTC Therapeutics’ sepiapterin for treating children and adults with a rare.

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Ultrasound of a baby
Drugs Health

Fetus treated with risdiplam for rare genetic disorder, a medical first

HQ Team February 27, 2025: A study published in The New England Journal of Medicine (NEJM) reveals that administration of the oral drug.

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Mirum Pharmaceuticals Inc.’s oral cholesterol drug for treating a rare metabolic disease has been approved by the Food and Drug Administration.
Drugs Health Medical

US regulator clears Mirum’s oral drug for reducing cholesterol deposits

Mirum Pharmaceuticals Inc.’s oral cholesterol drug for treating a rare metabolic disease has been approved by the Food and Drug Administration.

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AstraZeneca Plc’s drug Fasenra got approval from the European Union regulator for treating adult patients with a rare inflammation of blood vessels.
Drugs Health Pharma

AstraZeneca’s drug to treat rare blood vessel disease gets EU nod

AstraZeneca Plc’s drug Fasenra got approval from the European Union regulator for treating adult patients with a rare inflammation of blood vessels.

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