Updates

February 26, 2021February 26, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a ... Read more
February 25, 2021February 25, 2021
Firdapse Developer Catalyst Will Hold Rare Disease Day Event on Feb. 26

Firdapse (amifampridine) developer Catalyst Pharmaceuticals is supporting Rare Disease Day 2021 by sponsoring and participating in a virtual Facebook Live event on Friday, Feb. 26, ... Read more
February 23, 2021February 19, 2021
This Rare Disease Day, We’re Grateful for Those Who Came Before Us

It is humbling to know that we are the beneficiaries of those who have gone before us. We stand on the shoulders of those who ... Read more
February 22, 2021February 22, 2021
Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research ... Read more
February 22, 2021February 22, 2021
About the European LEMS Registry

Patient registries collect information about the health status of people with a certain disease. They are particularly important for rare diseases such as Lambert-Eaton myasthenic ... 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

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

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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

Paramedical Treatment for LEMS

NORD’s Caregiver Respite Program Continues Through Pandemic

Art Contest Salutes Creativity of Rare Disease Communities

After My Daughter’s Diagnosis, I Put Off Taking Care of Myself

Your Treatment Team for LEMS

Rare Skin Cancer Patient’s LEMS Symptoms Ease With Antibody Treatment

As a Parent, Determining When to Step in to Help Is a Challenge

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Fisher Syndrome Can Co-occur With Asymptomatic LEMS

Living Purposefully in Our New Normal

Recreational Therapy for LEMS Patients

Catching Glimpses of Goodness

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

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

Subscribe to our mailing list

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

Paramedical Treatment for LEMS

NORD’s Caregiver Respite Program Continues Through Pandemic

Cancer Found After Death of LEMS Patient with Rare Immune-mediated Disorder

Art Contest Salutes Creativity of Rare Disease Communities

After My Daughter’s Diagnosis, I Put Off Taking Care of Myself

Your Treatment Team for LEMS

Rare Skin Cancer Patient’s LEMS Symptoms Ease With Antibody Treatment

As a Parent, Determining When to Step in to Help Is a Challenge

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

Palliative Care for LEMS

Fisher Syndrome Can Co-occur With Asymptomatic LEMS

Emotional Responses to a LEMS Diagnosis

Living Purposefully in Our New Normal

Recreational Therapy for LEMS Patients

Case Study Describes Cancer Immunotherapy Opdivo Leading to LEMS

Catching Glimpses of Goodness