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 a non-profit organization based in Woodbridge, Connecticut, founded in 2017 to develop custom therapeutics aimed at treating individuals with rare genetic disorders, particularly muscular dystrophy. The organization focuses on creating effective, life-saving treatments specifically tailored for patients who are often told there are no available cures for their conditions. Through collaborations with renowned researchers and clinicians, as well as support from charitable donors, Cure Rare Disease strives to enhance research and provide crucial resources and information to families and advocates affected by these disorders. Their commitment is to empower patients in their fight for long, fulfilling lives despite their health challenges.

PathMaker Neurosystems is a neurotechnology company focused on developing non-invasive treatment solutions for neuromotor disorders. Their innovative platform targets neuronal hyperexcitability to address serious neurological conditions. Initially, the company is applying its multi-site, precision-targeted neuromodulatory approach to treat stroke and amyotrophic lateral sclerosis (ALS). PathMaker is in the process of manufacturing a non-invasive device specifically designed for treating post-stroke spasticity. This novel approach allows for the regulation of motor neuron excitability, providing patients with effective treatment options that do not require drugs or surgical interventions.

Myosana Therapeutics, Inc. is a biotechnology company focused on developing non-viral gene therapies aimed at treating muscle diseases, particularly Duchenne muscular dystrophy and other neuromuscular conditions. Founded in 2018 and based in Seattle, Washington, the company has created a novel gene delivery platform that specifically targets muscle cells to facilitate the production of proteins that are deficient due to genetic mutations. By delivering these proteins directly to the cell nuclei, Myosana Therapeutics seeks to slow the degeneration of skeletal muscles and reduce the impact of heart failure, ultimately improving the quality of life and longevity for affected individuals.

Locanabio, Inc., based in San Diego, California, is a biotechnology company specializing in RNA-targeted gene therapies. Founded in 2016, the company focuses on developing treatments for severe neurodegenerative, neuromuscular, and retinal diseases. Locanabio's platform is designed to modify disease-causing RNA, offering therapeutic candidates distinct from traditional DNA-targeted approaches. The company aims to address a spectrum of underserved diseases, including those affecting the nervous system, muscles, and eyes.

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.