dm+d

Unassigned

New Medicines

Refractory seizures in children with inherited mitochondrial disease (mitochondrial epilepsy)

Information

New molecular entity
Licence extension / variation
PTC Therapeutics

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes

Category

15-Lipoxygenase inhibitor (enzyme that regulates oxidative stress and inflammation response pathways). Taken three times a day.
Inherited mitochondrial epilepsy is a manifestation of mitochondrial disease estimated to affect approximately 11,000-13,000 children in the US, Europe, Japan and Latin America [1].
Refractory seizures in children with inherited mitochondrial disease (mitochondrial epilepsy)
Oral

Friedreich's ataxia in children and young adults

Information

Licence extension / variation
PTC Therapeutics
PTC Therapeutics

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

15-Lipoxygenase inhibitor (enzyme that regulates oxidative stress and inflammation response pathways). Taken three times a day.
Friedreich’s ataxia is a rare genetic neuromuscular disorder that causes progressive loss of coordination, muscle weakness, and fatigue. prevalence is about 1.8 per 100,000 in the UK [1,2].
Friedreich's ataxia in children and young adults
Oral