Muscular Dystrophy Association

All Wheels Up's purpose is to raise awareness for safer and more dignified accessible air travel through research and activism. Our solution is for those who use electric wheelchairs, as well as properly modified Manual Wheelchairs, to independently maneuver themselves onto the plane with dignity and safety.

Cure Rare Disease is an independent non-profit foundation located in Woodbridge, Connecticut, established in 2017. The foundation focuses on developing custom therapeutics for individuals with rare genetic disorders, particularly those facing muscular dystrophy. Through collaborations with leading researchers and clinicians, Cure Rare Disease creates effective, life-saving treatments tailored to the specific needs of patients. The organization also serves as a resource hub for families and advocates, providing critical information and support. By leveraging partnerships with charitable donors, the foundation aims to accelerate research and improve the quality of life for those diagnosed with conditions deemed untreatable.

PathMaker Neurosystems is a neurotechnology company focused on developing non-invasive treatments for serious neurological disorders, particularly neuromotor conditions. The company is advancing a platform that employs neuronal hyperexcitability suppression, utilizing a multi-site, precision-targeted neuromodulatory approach. Initially, PathMaker is applying this innovative technology to address issues related to stroke and amyotrophic lateral sclerosis (ALS). They are in the process of manufacturing a non-invasive device specifically designed to treat post-stroke spasticity. This novel approach enables the regulation of motor neuron excitability, allowing patients to receive effective treatment without the need for drugs or surgery.

Myosana Therapeutics, based in Seattle, Washington, focuses on developing non-viral gene therapies aimed at treating muscle diseases, particularly Duchenne muscular dystrophy and other neuromuscular disorders. The company has created a unique gene delivery platform that specifically targets muscle cells, facilitating the production of proteins that are deficient due to genetic mutations. By delivering these proteins directly to the cell nuclei, Myosana Therapeutics aims to slow skeletal muscle degeneration and heart failure, ultimately enhancing patients' quality of life and reducing the burden of these diseases. Founded in 2018, Myosana Therapeutics stands at the forefront of innovative approaches in the biotech sector.

Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.

Amicus Therapeutics, Inc. is a biotechnology company dedicated to the discovery, development, and commercialization of therapies for rare and orphan diseases. Founded in 2002 and headquartered in Cranbury, New Jersey, the company focuses on developing innovative treatments, including Galafold, an orally administered pharmacological chaperone for Fabry disease. Amicus is also advancing its pipeline with a Phase 3 clinical study of AT-GAA for Pompe disease. The company's research includes a range of first-in-class small molecules designed to treat lysosomal storage diseases by stabilizing misfolded enzymes in patients. This therapeutic approach potentially enhances the effectiveness of enzyme replacement therapies. Amicus Therapeutics has established collaborations with institutions such as Nationwide Children's Hospital and the University of Pennsylvania to further its research and development efforts.

Asklepios BioPharmaceutical is a biotechnology company focused on developing adeno-associated virus (AAV) gene therapies aimed at treating genetic disorders and rare diseases. Established in 2001 and headquartered in Research Triangle Park, North Carolina, the company possesses proprietary AAV technology and manufacturing capabilities, as well as a robust pipeline of clinical programs. Its pipeline includes clinical-stage therapies for Pompe disease and congestive heart failure, alongside a diverse array of preclinical candidates targeting neuromuscular and central nervous system diseases. Additionally, Asklepios has out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. The company’s gene therapy platform features Pro10, a proprietary cell line manufacturing process, complemented by an extensive library of AAV capsids and promoters, supporting advancements in genetic medicine through global research collaborations and innovative delivery methods.

Asklepios BioPharmaceutical is a biotechnology company focused on developing adeno-associated virus (AAV) gene therapies aimed at treating genetic disorders and rare diseases. Established in 2001 and headquartered in Research Triangle Park, North Carolina, the company possesses proprietary AAV technology and manufacturing capabilities, as well as a robust pipeline of clinical programs. Its pipeline includes clinical-stage therapies for Pompe disease and congestive heart failure, alongside a diverse array of preclinical candidates targeting neuromuscular and central nervous system diseases. Additionally, Asklepios has out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. The company’s gene therapy platform features Pro10, a proprietary cell line manufacturing process, complemented by an extensive library of AAV capsids and promoters, supporting advancements in genetic medicine through global research collaborations and innovative delivery methods.