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FDA Approves Sanofi Genzyme’s Nexviazyme for Treatment of Pompe Disease
On Aug. 6, the US Food and Drug Administration (FDA) granted accelerated marketing approval to avalglucosidase alfa (Nexviazyme) for the treatment of people 1 year of age and older living…
Tags: Drug Approval, Healthcare, Research, Research Advances
Dreaming of a good night’s sleep?
More than one-third of adults in the United States fail to get the seven to nine hours of sleep they need, reports the National Sleep Foundation. Children and teens require…
Tags: Healthcare
It Is OK to Have Mixed Feelings About Resuming Activities After Being Vaccinated
For more than a year we heard these messages: Wash your hands. Wear a mask. Stay six feet apart. Now, the Centers for Disease Control and Prevention (CDC) says people who are…
Tags: COVID-19, Healthcare, Research, Resources, Staying Active
Clinical Trial Alert: Phase 1b/2a Study of Descartes-08 in Adults with Generalized Myasthenia Gravis
Researchers at Cartesian Therapeutics are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 1b/2a clinical trial to evaluate safety, tolerability and manufacturing feasibility of the investigational…
Research Study Alert: Survey for Caregivers of Children with SMA to Study Treatment Decision Making
Researchers at Michigan Medicine at the University of Michigan are seeking caregivers of children living with spinal muscular atrophy (SMA) to participate in a survey about treatment decision making. Over the…
Tags: Caregiving, Clinical Trial Alert, Parenting, Research
FDA approves Octapharma’s Octagam® 10% for Treatment of Adults with Dermatomyositis
On July 20, the US Food and Drug Administration (FDA) granted approval to Octagam® 10% for the treatment of adult dermatomyositis, a rare immune-mediated inflammatory disease. Octagam® 10% is the first and only intravenous immunoglobulin (IVIg) approved…
Clinical Trial Alert: Phase 3 Study of Pamrevlumab (FG-3019) in Combination with Systemic Corticosteroids in Ambulatory Boys with DMD
Researchers at FibroGen are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial to evaluate efficacy of the investigational drug pamrevlumab (FG-3019) in combination…
Clinical Trial Alert: Phase 2 Study of Pitolisant for Treatment of Excessive Daytime Sleepiness in Adults with Myotonic Dystrophy, Type 1 (DM1)
Researchers at Harmony Biosciences are seeking adults living with myotonic dystrophy, type 1 (DM1) to participate in a phase 2 clinical trial and open-label extension to evaluate safety and efficacy of…
Clinical Trial Alert: Phase 1b and Open-Label Extension Study of ASP0367 in Boys with DMD
Researchers at Astellas Pharma Inc. are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 1b clinical trial and open-label extension to evaluate safety, tolerability and preliminary…
Spotting Neuromuscular Disease in Adults
After years of denial, it was the stairs to his second-floor college apartment that forced Chris Anselmo to finally face his limb-girdle muscular dystrophy (LGMD) in 2009. “I remember moving in, trying…
Tags: Healthcare, Research, Resources