All about Congenital Myasthenic Syndromes at a glance

Get essential information about congenital myasthenic syndromes (CMS) and why clinical trials are a crucial element in the path towards better treatment for the condition.

Get the basics on needle anxiety, one of the top medical fears among children, and discover tips to manage it.

On World Oral Health Day (March 20), we’re taking a closer look at what your oral health can tell you about your well-being.

Author Erin Paterson talks about Huntington’s Disease, family planning, and the healing power of sharing your story.

Breaking down the differences between observational studies and clinical studies for drug research.

Orit recently joined the Know Rare business development team with the goal of helping other caregivers and patients learn more about how to engage with clinical studies and other opportunities for support. Here’s how she is reframing attitudes towards life with a rare condition.

Spotlight on Trial Equity, an organization whose mission is to address the underrepresentation of diverse populations in clinical trials, ultimately advancing health equity.

The National Tay-Sachs & Allied Diseases Association (NTSAD), leader in the worldwide fight to treat and cure Tay-Sachs, Canavan, GM1 and Sandhoff diseases, is hosting the first-ever, ExternallyLed Patient-Focused Drug Development Meeting for GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases) on Thursday, February 15, 2024.

As we step into Rare Disease Month this February, it's an opportune time to celebrate resilience, foster connection, and empower ourselves within the rare disease community.

In his last post, author Chris Anselmo explored why it’s so hard to ask for help. Now, he shares his tips for how to do it with confidence.

An expert on adolescent health shares tips for families navigating substance use concerns alongside rare disease.

As we head into 2024, we’ve taken a moment to look back at some of the latest headlines in rare disease from the last year.

Hello, Adversity author Chris Anselmo delves into the common obstacles that make seeking help challenging, particularly for many people living with rare disease, and the transformative power of overcoming those mental blocks.

Edgewise’s investigational drug is a pioneering treatment for Duchenne and Becker muscular dystrophies.

It’s National Influenza Vaccination Week: find out why the flu vaccine should be a critical part of your winter to-do list.

Caitlin Eppes shares the inspiring story of The Avery Project, an initiative named after her daughter and dedicated to research of her rare genetic variant, and discusses how her family defied one-in-a-million odds to find a breakthrough in their diagnostic journey.

How writer Chris Anselmo confronts one of the most challenging yet persistent aspects of living with rare disease.

Ongoing clinical trials are paving the way for reducing steroid dependency and improving treatment options for various medical conditions.

As a pivotal diagnostic date approaches, a Rare Mom reflects on the complicated emotions associated with pregnancy.