Phase 3 Study Shows Burosumab is Superior than Traditional Therapies for X-Linked Hypophosphatemia (XLH)

At the 2019 annual meeting of The Endocrine Society, ENDO 2019, results from a recent study evaluating burosumab for the treatment of X-linked hypophosphatemia (XLH) were presented. This drug was…

Continue Reading Phase 3 Study Shows Burosumab is Superior than Traditional Therapies for X-Linked Hypophosphatemia (XLH)
A Noninvasive Stool Test Could Identify Cirrhosis In Nonalcoholic Fatty Liver Disease and Nonalcoholic Steatohepatitis
DarkoStojanovic / Pixabay

A Noninvasive Stool Test Could Identify Cirrhosis In Nonalcoholic Fatty Liver Disease and Nonalcoholic Steatohepatitis

Nonalcoholic fatty liver disease (NAFLD) is a condition in which the buildup of fat in the liver poses health concerns. Nonalcoholic Steatohepatitis (NASH) is a more severe, and more rare…

Continue Reading A Noninvasive Stool Test Could Identify Cirrhosis In Nonalcoholic Fatty Liver Disease and Nonalcoholic Steatohepatitis

An International Organization Dedicated to  Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals

Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…

Continue Reading An International Organization Dedicated to  Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals

Patient Worthy is Excited to Announce a New Partnership with The B.L.A.I.R. Connection!

A New Partnership Patient Worthy is honored and excited to announce our new partnership with The B.L.A.I.R. Connection. The B.L.A.I.R. Connection was founded by Grey Chapin who knows firsthand what…

Continue Reading Patient Worthy is Excited to Announce a New Partnership with The B.L.A.I.R. Connection!
The Department of Education’s 2020 Budget is Requesting Cuts to the Special Olympics and Special Education. What Does This Mean for Rare Patients?
Pexels / Pixabay

The Department of Education’s 2020 Budget is Requesting Cuts to the Special Olympics and Special Education. What Does This Mean for Rare Patients?

If you are an avid follower of the latest news, then you have probably heard about one the latest sensational headlines to come out of DC lately: the US Department…

Continue Reading The Department of Education’s 2020 Budget is Requesting Cuts to the Special Olympics and Special Education. What Does This Mean for Rare Patients?

New Potential Combination Therapy for Acute Myelogenous Leukemia has Received Breakthrough Therapy Designation

Acute Myelogenous Leukemia Acute myelogenous leukemia (AML) is a rare blood cancer whose prevalence increases with age. There are still extremely limited treatment options for AML and the five-year survival…

Continue Reading New Potential Combination Therapy for Acute Myelogenous Leukemia has Received Breakthrough Therapy Designation
Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial
DarkoStojanovic / Pixabay

Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial

According to a story from BioSpace, the biotechnology company Spruce Biosciences recently announced that they have achieved positive proof of concept data in the company's clinical trial. This is a…

Continue Reading Proof of Concept Results Achieved in Congenital Adrenal Hyperplasia Trial
Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone
DarkoStojanovic / Pixabay

Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company's Phase 3…

Continue Reading Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone
Gene Therapy Proves Effective in Treating AADC Deficiency in Trial
geralt / Pixabay

Gene Therapy Proves Effective in Treating AADC Deficiency in Trial

According to the publication over the weekend from San Francisco CBS affiliate KPIX, a groundbreaking clinical trial for those with aromatic l-amino acid decadboxylase (AADC) deficiency was undertaken at UCSF…

Continue Reading Gene Therapy Proves Effective in Treating AADC Deficiency in Trial