Tags Posts tagged with "rare disease"

rare disease

Hosted in part by The Transverse Myelitis Association (TMA)

Your Sunshine State Getaway The 2017 Annual MDF Annual Conference Registration fee: $200-$250 — covers all conference events (general and breakout sessions, two evening social receptions,...

Living with PBC: Learning Together For patients, family, healthcare professionals, and so many more! FEATURING: Certified PBC Nurse Educator, Jenny Walker, and a Patient Ambassador, Laurie Call 1-844-514-6294...

Living with PBC: Learning Together For patients, family, healthcare professionals, and so many more! FEATURING: Certified PBC Nurse Educator, Jennifer Blood, and a Patient Ambassador, Connie Call 1-844-514-6294...

TGIF Patient Worthians! This week we have promising results on promising results. First up is news on a Dravet syndrome treatment. Second is a Lupus Nephritis...

On June 21st, Targeted Oncology posted the results from a phase 2 clinical trial for chronic lymphocytic leukemia (CLL). The trial examined the effects of...

Onxeo has recently received the 10th unanimous recommendation from the Data Safety Monitoring Board to continue with the Phase 3 study (coined ReLive) of...

You're happily tucking your toddler in for her afternoon nap when she starts to panic. Where's her favorite blankie and stuffed penguin? A peaceful nap...

At any time of the day, in any hospital, a nurse is giving instructions to a parent, spouse, child, sibling or friend of a...

Xynomic Pharma, a United States-based oncology research and development company, announced at the end of last month that it had entered into a partnership...

$200 for members $230 for non-members There will be thought-leaders speaking, general and breakout sessions AND research updates!  

Living with PBC: Learning Together For patients, family, healthcare professionals, and so many more! FEATURING: Certified PBC Nurse Educator, Jenny Walker, and a Patient Ambassador, Nishele Call 1-844-514-6294 to...

Communication in the medical field doesn’t happen as much as it should. But, when it does, great discoveries often occur. The thing is, science is...

At 5 years old, Holton Jones was diagnosed with ROHHAD, or rapid-onset obesity with hypothalamic dysfunction, hypoventilation, and autonomic dysregulation. According to the Pensacola News...

The Danish biotechnology company, Orphazyme, announced in early May of this year that enrollment was completed for its lead study program for Niemann-Pick type...

In collaboration with the Hereditary Neuropathy Foundation (HNF), Patient Worthy held an essay contest for tickets to a Charcot-Marie-Tooth disease event. We asked members of the...

Support for Ofev® just keeps coming! Ofev® (nintedanib) was first approved by the U.S. FDA in 2014 for the treatment of idiopathic pulmonary fibrosis (IPF) after...

Results from the Phase 3 CASTOR trial from Janssen Pharmaceutical Companies were recently released, finding that Darzalex (daratumumab) was extremely effective at reducing the...

A new public cord blood donation center recently opened up in New York. This is the second of this type of facility to open...

Adyn’s Dream is a charity in Athens, Ohio. The organization provides live events that benefit families and their children that have received a diagnosis of...

In Toledo, Ohio an 8-year-old child is one of 100,000 Americans who is affected by sickle cell anemia. According to WNDU, Terrilonnae Chatman has sickle cell...

Due to a rare disease affecting many parts of her body, my oldest daughter, Kelley, saw a number of different doctors in a number...