Amicus Therapeutics Seeks Participants for Observational Pompe Disease Study

Researchers at Amicus Therapeutics are looking for participants with late-onset Pompe disease who are being treated with enzyme replacement therapy (ERT) to participate in an observational study. The study is designed to help researchers evaluate the change in muscle function and respiratory endpoints over approximately nine to 12 months. During that time, participants will have . . .

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Trial to Test Firdapse in MG Seeks Participants

Researchers are looking for people with myasthenia gravis (MG) to participate in a phase 3 clinical trial to evaluate the safety and effectiveness of the investigational drug amifampridine phosphate (brand name Firdapse). Effects of the drug will be assessed in individuals with muscle-specific kinase (MuSK) antibody positive MG and in a smaller group with acetylcholine . . .

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Trial to Test Drug Targeting Muscle Cramps in CMT Seeks Participants

Researchers are looking for people with Charcot-Marie-Tooth disease (CMT) to participate in a phase 2 clinical trial to evaluate the safety and effectiveness of the investigational drug FLX-787-ODT. FLX-787-ODT, under development by Flex Pharma, is taken in tablet form and is designed to reduce muscle cramps in adults with CMT. Trial length is approximately three . . .

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Trial to Test MG Drug UCB7655 Seeks Participants

Researchers are looking for people with myasthenia gravis (MG) to participate in a multinational phase 2A clinical trial to evaluate the safety, tolerability and effectiveness of the investigational drug UCB7665. UCB7665, under development by UCB Pharma, is administered via subcutaneous (under-the-skin) injection and is designed to reduce the severity of muscle weakness and fatigue in . . .

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Santhera Seeks Participants for DMD Study, Makes Available EAP and Online DMD Resource

With a focus on respiratory care in Duchenne muscular dystrophy (DMD), Santhera Pharmaceuticals has made available new resources for individuals with the disease. In addition, the company is seeking individuals to participate in a phase 3 clinical trial to test a respiratory drug in people with DMD. SIDEROS trial seeks participants Researchers are looking for . . .

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MDA and ALS ONE Work to Advance ALS Biomarker Research

MDA has awarded a human clinical trial grant totaling $750,000 to ALS ONE to explore the potential for a type of imaging called positron emission tomography (PET) to measure inflammation in the brain that could serve as a biomarker for ALS. ALS ONE is an alliance between four institutional leaders in ALS treatment development: Massachusetts . . .

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Researchers Seek Participants for CMS Trial

Researchers are looking for children and adults with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate (brand name Firdapse).

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Ezutromid Shows Potential to Treat DMD

Ezutromid

Summit Therapeutics plc has announced encouraging interim results from its phase 2 PhaseOut DMD clinical trial that suggest the MDA research grant-funded experimental treatment ezutromid (formerly SMT C1100) potentially may be effective as a treatment for DMD.

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