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 clinical-stage neurotechnology company that develops neuromodulation therapies for neuromotor disorders, including amyotrophic lateral sclerosis. The company focuses on non-invasive, multi-site direct current stimulation to modulate neural pathways and spinal motor neurons involved in disease processes. Its approach aims to reduce protein aggregation and motor neuron hyperexcitability, with the goal of improving motor function and potentially extending survival for ALS patients.

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 is a biotechnology company developing RNA-targeted gene therapies for underserved diseases, with a focus on neuromuscular, neurodegenerative, and retinal conditions. It employs an RNA-targeting platform to modify disease-causing RNA and correct aberrant transcripts, offering therapies distinct from DNA-based approaches. The company identifies target RNA sequences within transcripts and programs therapies to advance candidates for rare genetic neuromuscular, neurodegenerative, and retinal diseases. Located in San Diego, Locanabio aims to translate RNA-directed interventions into treatments for patients with severe conditions.

Amicus Therapeutics is a biotechnology company focused on discovering, developing, and delivering therapies for rare and orphan diseases. It specializes in oral pharmacological chaperones—small molecules that bind, stabilize, and enhance activity of misfolded enzymes—to treat lysosomal storage disorders such as Fabry and Pompe disease, and it explores combination approaches with enzyme replacement therapy. The company maintains a growing pipeline targeting additional lysosomal diseases, including Gaucher, and collaborates with academic and clinical partners to advance its programs. Amicus was founded in 2002 and is headquartered in Cranbury, New Jersey.

Asklepios BioPharmaceutical is a biotechnology company focused on developing adeno-associated virus (AAV) gene therapies for genetic disorders and other rare diseases. It employs proprietary AAV technology and a manufacturing platform to advance a diverse pipeline that includes in-house clinical programs in Pompe disease and congestive heart failure, as well as preclinical programs spanning neuromuscular and central nervous system conditions. The company also collaborates and out-licenses clinical indications such as hemophilia and Duchenne muscular dystrophy, supported by capabilities in scalable cell-line manufacturing and an extensive AAV capsid and promoter library. Founded in 2001 and based in Research Triangle Park, North Carolina, the company seeks to advance genetic medicines through collaborations, new delivery methods, and ongoing pipeline expansion.

Asklepios BioPharmaceutical is a biotechnology company focused on developing adeno-associated virus (AAV) gene therapies for genetic disorders and other rare diseases. It employs proprietary AAV technology and a manufacturing platform to advance a diverse pipeline that includes in-house clinical programs in Pompe disease and congestive heart failure, as well as preclinical programs spanning neuromuscular and central nervous system conditions. The company also collaborates and out-licenses clinical indications such as hemophilia and Duchenne muscular dystrophy, supported by capabilities in scalable cell-line manufacturing and an extensive AAV capsid and promoter library. Founded in 2001 and based in Research Triangle Park, North Carolina, the company seeks to advance genetic medicines through collaborations, new delivery methods, and ongoing pipeline expansion.