Rylee’s Story: A Cyclic Vomiting Syndrome Diagnosis Brings Support from the Community
source: pixabay.com

Rylee’s Story: A Cyclic Vomiting Syndrome Diagnosis Brings Support from the Community

  A correct diagnosis is often difficult to obtain, especially for a rare disease. Symptoms can be attributed to more common conditions, or they can be written off entirely. Rylee…

Continue Reading Rylee’s Story: A Cyclic Vomiting Syndrome Diagnosis Brings Support from the Community
Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1
source: pixabay.com

Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1

Genentech (Roche Group) announced positive results of its FIREFISH trial in a recent Business Wire news release.  The second part of the study evaluated risdiplam’s efficacy on infants ages one…

Continue Reading Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1

Blood Thinners Improves Quality of Life With Idiopathic Pulmonary Arterial Hypertension, Worsens Those with Other Variation

A medication that works for some patients with a rare disease may not be effective for others, even with the same disease. For rare diseases, this is important because sample…

Continue Reading Blood Thinners Improves Quality of Life With Idiopathic Pulmonary Arterial Hypertension, Worsens Those with Other Variation

Five Studies that Have Improved Care for Those with Inflammatory Bowel Disease

About three million Americans are affected by inflammatory bowel disease. Over the years there have been many improvements to treatment for this condition, but there are still further advances that…

Continue Reading Five Studies that Have Improved Care for Those with Inflammatory Bowel Disease
Acromegaly Community to Host Patient-Focused Drug Development Meeting with FDA
source: pixabay.com

Acromegaly Community to Host Patient-Focused Drug Development Meeting with FDA

On April 28th, 2020, Acromegaly Community, a nonprofit organization that is dedicated to supporting acromegaly patients, will be hosting a patient focused drug development meeting (PFDD). These meetings are an…

Continue Reading Acromegaly Community to Host Patient-Focused Drug Development Meeting with FDA
Close Menu