Updates

December 4, 2020December 4, 2020
MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine

The Muscular Dystrophy Association (MDA) is encouraging the U.S. Centers for Disease Control and Prevention (CDC) to recommend that people living with neuromuscular diseases ... Read more
December 3, 2020December 3, 2020
NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the ... Read more
November 30, 2020November 25, 2020
We’re Staying Home for the Holidays

Christmas is by far my favorite time of the year. Holiday music, trimming the tree, baking cookies, family gatherings — none of this can ... Read more
November 30, 2020November 30, 2020
FAQs About Firdapse as Treatment for LEMS

Firdapse is an approved treatment for patients ages 17 and older with Lambert-Eaton myasthenic syndrome or LEMS. How does Firdapse work? Firdapse works by ... Read more
November 26, 2020November 26, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their ... Read more

Subscribe to our mailing list

Get regular updates delivered to your email box.

Thanks! Please check your email for a confirmation email.

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 ... Read more

Updates

MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

FAQs About Firdapse as Treatment for LEMS

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The Job I Never Wanted

How to Prepare for IVIG When You Have LEMS

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

FAQs About IVIG for LEMS

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

Rare Case of Lung Cancer-linked LEMS Underscores Importance of Screening

Subscribe to our mailing list

Sleep Hygiene for LEMS Patients

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Firdapse Now Widely Available in Canada for Treating LEMS, KYE Pharmaceuticals Says

Brain Fog in LEMS

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Catalyst Given US Patent Covering Firdapse in Treating LEMS

Tips for LEMS Caregivers

NSF Funds Project to Make Better Computer Simulation of Nerve-muscle Communication

Independent Living for People With LEMS

Newly Funded UNC Computer Simulations Could Help Find LEMS Treatments

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

How to Help a Family Member With LEMS

Ruzurgi Now Available in Canada for Patients Ages 6 or Older

How a Concussion Led to a Near-death Experience

Will LEMS Affect my Sex Life?

Planning for the Future After a LEMS Diagnosis

My Concussion Worsened Instead of Getting Better

Raising Awareness of LEMS

Wait Between Repeat Nerve Stimulation Tests in LEMS, Researcher Advises

Same But Different Photo Contest Celebrates People With Rare Diseases

Catalyst, KYE Agree to Commercialize Firdapse for Adult LEMS Patients in Canada

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

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

MDA Wants Neuromuscular Disease Patients to Have Early Access to COVID-19 Vaccine

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

We’re Staying Home for the Holidays

FAQs About Firdapse as Treatment for LEMS

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

The Job I Never Wanted

How to Prepare for IVIG When You Have LEMS

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

FAQs About IVIG for LEMS

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

Rare Case of Lung Cancer-linked LEMS Underscores Importance of Screening

Subscribe to our mailing list

Sleep Hygiene for LEMS Patients

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Firdapse Now Widely Available in Canada for Treating LEMS, KYE Pharmaceuticals Says

Brain Fog in LEMS

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

Catalyst Given US Patent Covering Firdapse in Treating LEMS

Tips for LEMS Caregivers

NSF Funds Project to Make Better Computer Simulation of Nerve-muscle Communication

Independent Living for People With LEMS

Newly Funded UNC Computer Simulations Could Help Find LEMS Treatments

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

How to Help a Family Member With LEMS

Ruzurgi Now Available in Canada for Patients Ages 6 or Older

How a Concussion Led to a Near-death Experience

Will LEMS Affect my Sex Life?

Planning for the Future After a LEMS Diagnosis

My Concussion Worsened Instead of Getting Better

Raising Awareness of LEMS

Wait Between Repeat Nerve Stimulation Tests in LEMS, Researcher Advises

Same But Different Photo Contest Celebrates People With Rare Diseases

Catalyst, KYE Agree to Commercialize Firdapse for Adult LEMS Patients in Canada

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input