Research

Focus on the pharmaceutical and biotech industry
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).

Hemophilia Explained

Hemophilia Explained

Joe Pugliese of the Hemophilia Alliance, a not-for-profit organization of federally funded hemophilia treatment centers that is working to ensure its member have the expertise, resources and public support to sustain their integrated clinical and pharmacy services for persons with hemophilia and other bleeding disorders.

Gene Therapy for Hurler Syndrome

Gene Therapy for Hurler Syndrome

Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics, discussuses his company’s gene therapy being developed for MPS I.

Huntington’s Disease Drug Development and Patient Groups

Huntington’s Disease Drug Development and Patient Groups

Scott Schobel, MD, MSc, clinical science leader for Roche’s Huntington Disease Program talks about the role that Huntington disease patient group, throughout the world, helped Roche develop their clinical program focused on Huntington disease.

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