Sleep Quality Decreased in DS Caregivers
Soticlestat Associated With Sustained Seizure Reductions in LGS and DS
Soticlestat May Be Useful in LGS and DS Care
Pharmaceutical CBD May Be Beneficial in LGS and DS Management
Latest Clinical Trials
Features & Insights
The Expanding Power of Precision Medicine in Treating Lennox-Gastaut Syndrome
Precision medicine aims to provide customized and individualized medical treatment for LGS based on a patient’s unique characteristics.
As Pandemic Restrictions Continue to Ease, Rare Disease Patients Still Fear COVID-19
Three years after COVID-19 was declared a pandemic, rare disease patients still fear the contagion—and must deal with the fallout.
At 40, Orphan Drug Act Enjoys Rare Bipartisan Support
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
Drug Repurposing: A Novel Strategy for a Deeply Unequal World
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.
Exploring the Parental Burden of Care of Children With Rare Diseases
The parental burden of care of children with rare diseases is considerable and deserves due attention and support.
EveryLife Foundation Navigates Complex Issues to Advocate for Rare Disease Patients
The EveryLife Foundation for Rare Diseases pushes a legislative agenda aimed at benefitting the 30 million Americans with a rare disease.
Synthetic Control Arms Could Facilitate More RCTs in Rare Disease
Dr. Rahul Banerjee of the University of Washington discusses the benefits and drawbacks of using synthetic control arms in randomized controlled trials.