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Harmony Biosciences Holdings, Inc. Announces Positive Topline Data from Phase 2 Signal Detection Study Evaluating Pitolisant in Adult Patients with Myotonic Dystrophy Type 1

Harmony Biosciences Holdings, Inc. announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy. ....

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Pepgen Reports Third Quarter 2023 Financial Results And Recent Corporate Developments

Pepgen Reports Third Quarter 2023 Financial Results And Recent Corporate Developments
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PepGen (PEPG) Announces FDA has Lifted the Clinical Hold on its INDA for FREEDOM-DM1 Phase 1 Study of PGN-EDODM1

PepGen (PEPG) Announces FDA has Lifted the Clinical Hold on its INDA for FREEDOM-DM1 Phase 1 Study of PGN-EDODM1
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Avidity Biosciences Announces New Positive AOC 1001 Data Demonstrating Improvement in Multiple Additional Functional Endpoints and Favorable Long-term Safety and Tolerability in People with Myotonic Dystrophy Type 1

New AOC 1001 data demonstrate improvement in additional functional measures including hand grip, muscle strength and patient reported outcomes, augmenting previously reported positive data showing. ....

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Avidity Biosciences Announces New Positive AOC 1001 Data Demonstrating Improvement in Multiple Additional Functional Endpoints and Favorable Long-term Safety and Tolerability in People with Myotonic D

Avidity Biosciences Announces New Positive AOC 1001 Data Demonstrating Improvement in Multiple Additional Functional Endpoints and Favorable Long-term Safety and Tolerability in People with Myotonic D
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