Myotonic Dystrophy Type 1 (MD1)

Myotonic Dystrophy Type 1 (MD1)

by | May 7, 2021

Therapeutic area: Neuromuscular Disorders Disease: Myotonic Dystrophy Type 1 (MD-1) Monogenic inroad: DMPK Cellular Model: Patient derived skeletal muscle cells Project Origin: I-Stem,  Cécile Martinat and Prof Marc Peschanski  Medical need: MD1 is a type of muscular...
Dilated Cardio Myopathy (DCM)

Dilated Cardio Myopathy (DCM)

by | May 6, 2021

Therapeutic area: Muscular Disorders, Cardiomyopathy Disease: Dilated Cardio Myopathy  Monogenic inroad: TITIN Cellular Model: Patient-derived cardiomyocytes (ventricular) Project Origin:   Technische Universität München, Klinikum rechts der Isar, Prof. Karl-Ludwig...
Myofibrillar Myopathy

Myofibrillar Myopathy

by | Mar 31, 2021

Therapeutic area: Muscular Disorders, Cardiomyopathy Disease: Myofibrillar Myopathy Monogenic inroad: Desmin Cellular Model: Cardiomyocytes Project Origin: Sorbonne University, Prof. Onnik Agbulut Medical need: Myofibrillar myopathies are a group of rare genetic...

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