by Peter Ciszewski | Aug 18, 2021
Michael J. Thorpy, MD, ChB, Director of the Sleep-Wake Disorders Center at the Montefiore Medical Center, discusses the post hoc analyses of data from the REST-ON clinical trial of FT218, an investigational treatment for narcolepsy. Narcolepsy is a rare...
by Peter Ciszewski | Aug 17, 2021
Wes Michael, President and Founder of Rare Patient Voice, discusses the company’s main objectives as well as a recent milestone they have reached. As Mr. Michael explains, Rare Patient Voice is a market research company. Their goal is to give rare disease...
by Peter Ciszewski | Aug 16, 2021
Omar Sinno, MD, Medical Lead of Rare Disease at UCB, describes the current standard of care for myasthenia gravis. Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include...
by Peter Ciszewski | Aug 13, 2021
Tracy Dixon-Salazar, PhD, Executive Director of the Lennox-Gastaut Syndrome (LGS) Foundation, discusses the disorder’s unique resistance to treatment. LGS is a rare neurological condition that usually begins in childhood. It is characterized by multiple types...
by Peter Ciszewski | Aug 12, 2021
Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics, discusses why diagnosis of Friedreich’s ataxia can be delayed and how the disease tends to progress. Friedreich’s ataxia is a degenerative neuromuscular disorder caused by mutations in the FXN gene...
