Updates

April 13, 2021April 13, 2021
Patients, Caregivers, Loved Ones Sought for Survey

The Mighty, a digital health community for people with health challenges and disabilities, is urging Lambert-Eaton myasthenic syndrome (LEMS) patients, their caregivers, and loved ones to ... Read more
April 12, 2021April 7, 2021
We Recognized the Great Value in Community During the Pandemic

We are living in unprecedented times involving a pandemic and political and social unrest that has been debated in many homes and communities. Even amid ... Read more
April 6, 2021April 6, 2021
Report Examines Pros, Cons of Orphan Drug Status

The Orphan Drug Act (ODA) has spurred the development of treatments for rare diseases, such as Lambert-Eaton myasthenic syndrome (LEMS), according to a recent report. ... Read more
April 5, 2021March 31, 2021
How Books Inspire Us to Embrace Our Circumstances

The paths of those affected by Lambert-Eaton myasthenic syndrome (LEMS) hold many twists and turns. Our family has experienced many ups and downs since our ... Read more
March 30, 2021March 30, 2021
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how ... Read more

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June 4, 2018June 4, 2018

FDA Accepts Catalyst’s NDA for Firdapse with Priority Review Status for LEMS

News

The U.S. Food and Drug Administration has accepted Catalyst Pharmaceuticals’ new drug application (NDA) for Firdapse (amifampridine phosphate) for Lambert-Eaton myasthenic syndrome (LEMS), with priority review status. Firdapse is a formulation of amifampridine phosphate in development for the treatment of LEMS as well ... Read more

Updates

Patients, Caregivers, Loved Ones Sought for Survey

We Recognized the Great Value in Community During the Pandemic

Report Examines Pros, Cons of Orphan Drug Status

How Books Inspire Us to Embrace Our Circumstances

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

I Acknowledge the Sadness and Then Count My Blessings

Development of Long-acting Firdapse on Track, Catalyst Says

The Fog of Uncertainty Lifted When My Daughter Was Finally Diagnosed

Spending on Rare Disease Treatments Such as LEMS Comparatively Low

Motion Analysis Helped Grace Get the Most Out of Physical Therapy

Acknowledging the Loss From Chronic Illness Helped Us Emotionally Heal

Subscribe to our mailing list

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

Equine-assisted Therapy for LEMS

MedicAlert, NORD Partner to Help Patients in Emergencies

How Our Family Is Fighting Depression as Quarantine Continues

About the Firdapse Pregnancy Registry for LEMS Patients

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

This Rare Disease Day, We’re Grateful for Those Who Came Before Us

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

About the European LEMS Registry

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Our Daughter Is Seeing Positive Results From Rituxan

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Connecting With Our Children in Meaningful Ways

Hydrotherapy for LEMS

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Equine-assisted Activities Have Restored Joy in Our Daughter’s Life

Massage Therapy for LEMS

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

LEMS Treatments Don’t Affect Calcium Channels, Study Finds

Weighing the Pros and Cons of My Daughter’s IVIG Therapy

FDA Accepts Catalyst’s NDA for Firdapse with Priority Review Status for LEMS

Patients, Caregivers, Loved Ones Sought for Survey

We Recognized the Great Value in Community During the Pandemic

Report Examines Pros, Cons of Orphan Drug Status

How Books Inspire Us to Embrace Our Circumstances

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

I Acknowledge the Sadness and Then Count My Blessings

Development of Long-acting Firdapse on Track, Catalyst Says

The Fog of Uncertainty Lifted When My Daughter Was Finally Diagnosed

Spending on Rare Disease Treatments Such as LEMS Comparatively Low

Motion Analysis Helped Grace Get the Most Out of Physical Therapy

Acknowledging the Loss From Chronic Illness Helped Us Emotionally Heal

Subscribe to our mailing list

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

Equine-assisted Therapy for LEMS

MedicAlert, NORD Partner to Help Patients in Emergencies

How Our Family Is Fighting Depression as Quarantine Continues

About the Firdapse Pregnancy Registry for LEMS Patients

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Firdapse Developer Catalyst Will Hold Rare Disease Day Event on Feb. 26

This Rare Disease Day, We’re Grateful for Those Who Came Before Us

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

About the European LEMS Registry

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Our Daughter Is Seeing Positive Results From Rituxan

Importance of Advocacy for LEMS Patients

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Connecting With Our Children in Meaningful Ways

Hydrotherapy for LEMS

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Equine-assisted Activities Have Restored Joy in Our Daughter’s Life

Massage Therapy for LEMS

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

LEMS Treatments Don’t Affect Calcium Channels, Study Finds

Weighing the Pros and Cons of My Daughter’s IVIG Therapy