Muscle Weakness Articles & Analysis
11 news found
CD Genomics, a leading biotechnology company specializing in genomics and sequencing, has announced the launch of its advanced Mitochondrial Diseases Panel Sequencing services. This new panel enables rapid and accurate detection of mutations in mitochondrial DNA (mtDNA) that are associated with a wide range of mitochondrial disorders. Mitochondrial diseases are a complex and heterogeneous group ...
Clinical data demonstrating initial safety and efficacy from the RESKUE trial are being presented by Chief Medical Officer Maria Escolar, M.D., at the 29th Congress of European Society of Gene & Cell Therapy (ESGCT), October 11-14, 2022 Subjects treated with FBX-101 have shown increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid (CSF), normal white matter ...
FBX-101 clinical data demonstrating safety and initial efficacy from the RESKUE trial is being presented at the 2022 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium FBX-101 is a systemic AAV gene therapy following unrelated cord blood transplantation (“UCBT”) and has been well tolerated through Day 180 FBX-101 significantly increased GALC enzyme ...
Every year, the month of August is nationally recognized as Spinal Muscular Atrophy (SMA) Awareness Month. This month-long awareness campaign serves as an important time to demonstrate support and help promote awareness for this rare disease affecting lives across the world. WHAT IS SMA? Affecting 4 people in every 100,000 in the U.S., Spinal Muscular Atrophy (SMA) is a genetic, progressive ...
FireGene announces that its Monkeypox Virus Nucleic Acid Detection Kit (Real-time Fluorescent PCR Method) has recently obtained CE-IVDD approval. This RT-PCT kit can be used to detect suspected cases of MPV infection. Rash fluid, nasopharyngeal swabs, pharyngeal swabs, and serum samples are applicable for the test. The test result is available in 40 minutes after sample collection. This kit is ...
ByFireGene
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in a mouse model of infantile onset Pompe disease. The results, which will be presented today during the “Disease models and Clinical Applications: ...
The narcolepsy drug Wakix can now be prescribed to patients in China with urgent unmet medical needs BOSTON, Mass., and Boao, Hainan, China, May 07, 2021 – Citrine Medicine, a China-based rare disease therapeutics company, today announced that the narcolepsy therapy Wakix® (pitolisant) has been approved by the Health Commission and Medical Products Administration of Hainan Province ...
Applying non-invasive electrical stimulation to the expiratory abdominal muscles may reduce abdominal muscle atrophy and markedly reduce the number of days adult patients require mechanical ventilation – a potentially important finding for weaning ventilated patients, including seriously ill COVID-19 patients, and improving patient outcomes. Liberate Medical today announced the results of ...
TIKOMED, a Swedish privately-held biopharmaceutical company today announced that the European Commission has granted Orphan Drug Designation to its lead drug candidate ILB® for the treatment of patients with amyotrophic lateral sclerosis (ALS) – a terminal, neurodegenerative disease where most patients die of respiratory muscle weakness in less than three to five years from symptom ...
Heat waves are the deadliest of extreme weather events, the Centers for Disease Control and Prevention reported this week. A case study published in the August 9 edition of the agency’s journal, Morbidity and Mortality Weekly Report, looked at heat illness in New York City from 2000 to 2011. The results revealed that there were more than 150 deaths, about 1,600 hospitalizations and around ...
Liberate Medical, a clinical stage medical device company, focused on the development of novel non-invasive neuromuscular electrical stimulation devices to address pulmonary conditions, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Device Designation for the company’s VentFree muscle stimulator. The proposed indication for the device is preventing ...