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PTC Presents Results from a Real-World Study of Steroid Switching in the Treatment of Patients with Dystrophinopathies

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to EMFLAZA® (deflazacort) following the FDA approval. The primary reason the majority of patients chose to switch to EMFLAZA was a desire to delay disease progression to improve the benefit and tolerability. During the 6-months average follow-up after switching, physician-reported outcomes were consistent with EMFLAZA addressing the primary reasons for switching. Data were presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference. ....

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Axon Therapies Announces First Patient Treated In The Rebalance-HF IDE Feasibility Trial

Axon Therapies Announces First Patient Treated In The Rebalance-HF IDE Feasibility Trial
yahoo.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from yahoo.com Daily Mail and Mail on Sunday newspapers.

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Clene Nanomedicine Presents Blinded Interim Results from RESCUE-ALS Phase 2 Study at the 31st International Symposium on ALS/MND

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SALT LAKE CITY, Dec. 10, 2020 (GLOBE NEWSWIRE) Clene Nanomedicine, Inc., a clinical-stage biopharmaceutical company, today announced the presentation of blinded interim results from the Phase 2 RESCUE-ALS clinical trial investigating the effects of its lead clinical candidate, CNM-Au8, for the treatment of amyotrophic lateral sclerosis (ALS). CNM-Au8 is an aqueous suspension of clean-surfaced, faceted gold nanocrystals with catalytic activity that has been shown to enhance the metabolic energetic capacity of motor neurons while simultaneously reducing oxidative stress.
As of the data cutoff (October 27, 2020), the trial was fully enrolled with a preliminary blinded assessment of the study s primary endpoint, the motor neuron number index-4 [MUNIX(4)] score, showing that more than 40% of enrolled patients with completed week 12 data experienced improvements in motor neuron function assessed by MUNIX. When compared to baseline values, the average MUNIX(4) score o ....

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