Orphan Diseases Companies and Organizations

 

10 Organization(s)

Acer Therapeutics

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Acer Therapeutics is developing therapies with established clinical proof-of-concept for the treatment of serious, ultra-rare diseases with critical unmet medical need. We are committed to ...

AGADA Biosciences

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AGADA facilitates orphan drug development with robust pre-clinical efficacy studies and clinical study support.

 

AOP Orphan Diseases

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Orphan disease treatments

Dynacure

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Dynacure is a biotechnology company developing new treatments for patients affected by serious orphan disorders. 

Emmaus

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Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan disease ...

Inventiva

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Inventiva is a biopharmaceutical company with several drug candidates at clinical and preclinical stage whose objective is to develop and provide patients with new therapies. The focus of the ...

Palladio Biosciences

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Palladio Biosciences, Inc, a private biotech company, was founded in 2015 to develop transformative medicines for orphan diseases of the kidney.

Qrativ

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Qrativ uses the power of combining the nference artificial intelligence (AI)-driven knowledge synthesis platform with Mayo Clinic’s medical expertise and clinical ...

Rhythm Pharma

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Rhythm is a biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic ...

Santhera Pharmaceuticals

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Orphan Neuromuscular Disease Targets
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