Dravet Syndrome (DS)

Features & Insights

LGS Features

The Expanding Power of Precision Medicine in Treating Lennox-Gastaut Syndrome

By

Kyle Habet, MD

Publish Date May 23, 2023

Precision medicine aims to provide customized and individualized medical treatment for LGS based on a patient’s unique characteristics.

Pompe Features

As Pandemic Restrictions Continue to Ease, Rare Disease Patients Still Fear COVID-19

By

Larry Luxner

Publish Date April 12, 2023

Three years after COVID-19 was declared a pandemic, rare disease patients still fear the contagion—and must deal with the fallout.

Features

At 40, Orphan Drug Act Enjoys Rare Bipartisan Support

By

Larry Luxner

Publish Date January 18, 2023

Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.

ALGS Insights

Drug Repurposing: A Novel Strategy for a Deeply Unequal World

By

Ryner Lai, MBBS

Publish Date December 12, 2022

The belief driving drug repurposing is that the current drugs that we do have may yet possess therapeutic aspects we aren’t fully aware of.

HAE Insights

Exploring the Parental Burden of Care of Children With Rare Diseases

By

Ryner Lai, MBBS

Publish Date December 8, 2022

The parental burden of care of children with rare diseases is considerable and deserves due attention and support.

Features

EveryLife Foundation Navigates Complex Issues to Advocate for Rare Disease Patients

By

Larry Luxner

Publish Date November 21, 2022

The EveryLife Foundation for Rare Diseases pushes a legislative agenda aimed at benefitting the 30 million Americans with a rare disease.

Features

Synthetic Control Arms Could Facilitate More RCTs in Rare Disease

By

Tori Rodriguez, MA, LPC, AHC

Publish Date November 14, 2022

Dr. Rahul Banerjee of the University of Washington discusses the benefits and drawbacks of using synthetic control arms in randomized controlled trials.