Congenital myasthenic syndrome with episodic apnea Archives

CMS is so Rare That it Took Four Years to Enroll Twenty People for a Trial

Post author:Rose Duesterwald
Post published:November 4, 2019
Post category:Congenital myasthenic syndrome with episodic apnea Lambert-Eaton Myasthenic Syndrome

According to a recent article in Biospace, Catalyst Pharmaceutical’s CMS-001 Phase 3 study ended with mixed results. The trial was billed as the first placebo-controlled double-blind study testing amifampridine phosphate (brand…

We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.

Our goals are to share stories, cultivate strong community, provide the latest medical findings, connect people and pioneer production of patient worthy information. Help us attain these goals by telling us a little bit about yourself!

CMS is so Rare That it Took Four Years to Enroll Twenty People for a Trial

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

“Shades of Psoriasis”: Plaque Psoriasis and Sharing Your Story

Understanding RSV: Why Combatting Ethnic and Age-Related Health Disparities Could Improve Outcomes

We Need Equity and Diversity Within Rare Disease Research

DNA in Color: How Ronya Nelson is Working to Close the Diversity Gap in Genetics - Part 1

Bridging the Diversity Gap in Healthcare

Congratulations to the 2023 Recipients of the Diversity in Lupus Research Awards!

Welcome to Study of the Week. We go in-depth and select a study we think is of particular interest, discussing details, explaining its importance, who may be impacted and lots more!

Let’s Work Together!

Keep Up to Date

Learn More