Research

Focus on the pharmaceutical and biotech industry
Diagnosing AADC Deficiency

Diagnosing AADC Deficiency

  Claudio Santos, MD, MBA, of PTC Therapeutics describe the pathophysiology of Aromatic l-amino acid decarboxylase (AADC) deficiency and how it is diagnosed. AADC deficiency is a rare genetic disorder due to mutations in the DDC gene that regulates the AADC...

Gene Control vs Gene Therapy

Gene Control vs Gene Therapy

David Roth, MD of Syros Pharmaceuticals describes his company’s gene control methodology to target genetic conditions in a unique way.

Thyroid Eye Disease: Incidence and Treatment

Thyroid Eye Disease: Incidence and Treatment

Gary Joseph Lelli, MD, of the Department of Ophthalmology at Weill Cornell Medicine talks about the incidence and treatment options for thyroid eye disease due to Graves’ disease.

An Oral Film to Treat Lennox-Gastaut Disease

An Oral Film to Treat Lennox-Gastaut Disease

Daniel Barber is chief operating officer at Aquestive Therapeutics, a specialty pharmacy company that is highly focused on providing treatment options for persons unable to properly swallow medicine (e.g., people with epilepsy, ALS, Parkinson’s, etc).

Gene Therapy for ADA-SCID

Gene Therapy for ADA-SCID

Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics talks about his company’s gene therapy in development to treat children with adenosine deaminase severe combined immunodeficiency (ADA-SCID).

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