Norah Needs You: Help This Young Girl Find a Bone Marrow Donor
At four years old, Norah Gratz-Lazarus of California is a sweet, bubbly girl, full of spirit. She's a big sister, and loves jokes, ice cream, and chocolate chip cookies. However, Norah…
<a href="https://pixabay.com/users/frolicsomepl/">frolicsomepl</a> / Pixabay
Potential Treatment for Lupus is Granted Fast Track Designation From the FDA
According to a story from BioSpace, Incyte Corporation and Eli Lilly and Company recently announced that the US Food and Drug Administration (FDA) has given the drug baricitinib Fast Track…
<a href="https://pixabay.com/users/skeeze/">skeeze</a> / Pixabay
After Being Ignored by Doctors, Former NFL Cheerleader Accurately Diagnoses Own Pectus Excavatum
According to a story from NTD, a former NFL cheerleader successfully diagnosed her own rare disease, pectus excavatum, after her symptoms were dismissed by her doctors as nothing more than…
<a href="https://pixabay.com/users/Counselling/">Counselling</a> / Pixabay
Sexism and Misdiagnosis Led to Years of Suffering for This Chronic Fatigue Syndrome Patient
According to a story from Cosmopolitan, a young woman who we will call Courtney (real name changed for privacy) was diagnosed with chronic fatigue syndrome. However, this diagnosis only occurred…
<a href="https://pixabay.com/users/Pexels/">Pexels</a> / Pixabay
Markelle Fultz of the Philadelphia Sixers was Diagnosed With Thoracic Outlet Syndrome. What Does This Mean?
According to a story from philly.com, Markelle Fultz, who plays as a guard for the Philadelphia 76ers, was recently diagnosed with thoracic outlet syndrome. It has not been good news…
<a href="https://pixabay.com/users/1041483/">1041483</a> / Pixabay
New Measure Developed to Assess the Severity of Charcot-Marie-Tooth Disease in Babies
According to a story from Charcot-Marie-Tooth News, a group of scientists have developed a new scale that can be used to predict disease severity in infants that are diagnosed with…
<a href="https://pixabay.com/users/PixLoger/">PixLoger</a> / Pixabay
Phase 1 Gene Transfer Trial for Recessive Dystrophic Epidermolysis Bullosa is Recruiting Patients
According to a story from Debra of America, a new Phase 1 clinical trial being operated by Stanford University is currently recruiting participants. This trial will be a test of…
<a href="https://pixabay.com/users/webandi/">webandi</a> / Pixabay
Parents Mourn the Loss of Six Month Old Daughter to Mitochondrial Disease
According to a story from Cornwall Live, parents Ben Keverne and Hannah Chapman were recently devastated by the loss of their daughter Maisie. The little girl was diagnosed with mitochondrial…
<a href="https://pixabay.com/users/DarkoStojanovic/">DarkoStojanovic</a> / Pixabay
Scientists May Have Found a New Way to Predict The Severity of Type 2A Charcot-Marie-Tooth Disease
According to a story from Charcot-Marie-Tooth news, researchers have found that models of mutations affecting the mitofusin 2 protein (MFN2), which is the protein that is affected Charcot-Marie-Tooth disease type…
Charcot-Marie-Tooth: Misconceptions from People Who Just Don’t Know
Charcot-Marie-Tooth disease (CMT), is a rare neurological condition. It's hereditary, and it affects the way nerves communicate with muscles. There are different types of CMT, determined by which genes are…
<a href="https://pixabay.com/users/HG-Fotografie/">HG-Fotografie</a> / Pixabay
Possible Therapeutic Target Identified For Silicosis
According to a story from bioengineer.org, a team of researchers from around the world have collaborated and successfully discovered a critical mechanism that leads to lung inflammation following exposure to…
<a href="https://pixabay.com/users/valelopardo/">valelopardo</a> / Pixabay
The Vital Role of the National Amyotrophic Lateral Sclerosis (ALS) Registry
According to a story from the Centers for Disease Control and Prevention, the National ALS Registry is playing a vital role in learning more about amyotrophic lateral sclerosis, a devastating…
<a href="https://pixabay.com/users/jarmoluk/">jarmoluk</a> / Pixabay
A Mother With Deadly Glioblastoma is Using Her Remaining Time to Make Memories With Her Kids
According to a story from liverpoolecho.co.uk, Isabel Morison is a 39-year-old music teacher with a nine year old son Dylan and a six year old daughter, Lucy. Last March, she…
<a href="https://pixabay.com/users/Free-Photos/">Free-Photos</a> / Pixabay
An Innovative UK Charity Helps a Woman With Chronic Fatigue Syndrome get Employed for the First Time
According to a story from Able Magazine, 37 year old Victoria Clutton recently got her first job thanks to the efforts of the UK nonprofit organization ASTRiiD (Available Skills for Training,…
NIH Apologizes for Censorship of Rare Disease Artwork
We wrote back in 2015 about Beyond the Diagnosis, a traveling art exhibit featuring portraits of children with rare diseases curated by the Rare Disease United Foundation. Its latest stop was…
<a href="https://pixabay.com/users/bodymybody/">bodymybody</a> / Pixabay
Research Reveals Possible Biomarkers for Serrated Colorectal Cancer
According to a story from Newswise, scientists have previously discovered that there are two pathways that colorectal polyps may utilize as they transform into colorectal cancer. These are dubbed the…
Hereditary Angioedema Discovery is First Step Toward Gene Therapy
Hereditary Angioedemia (HAE) results when the blood is missing the C1-inhibitor protein. It causes unpredictable and sudden attacks of swelling on various parts of the body. It can affect practically…
Editor’s Choice: Painters, Warriors, and Presidents of the Rare Community
Happy Wednesday! With Hanukah wrapped up, and Christmas just around the corner, things are getting pretty crazy. Take a second to relax and check out these four stories. First, we…
<a href="https://pixabay.com/users/sasint/">sasint</a> / Pixabay
Experimental Treatment for Ewing’s Sarcoma Earns Rare Pediatric Disease Designation From the FDA
According to a story from BioSpace, the cancer drug company Gibson Oncology, LLC has recently announced that the US Food and Drug Administration (FDA) has awarded Rare Pediatric Disease designation…
<a href="https://pixabay.com/users/ColiN00B/">ColiN00B</a> / Pixabay
Drug Developer Receives $900,000 for the Development of a Gene Therapy for Epidermolysis Bullosa
According to a story from pm360online.com, the gene therapy company Fibrocell Science, Inc., recently received an investment of $900,000 from the Epidermolysis Bullosa Medical Research Foundation (EBMRF) and the EB…
<a href="https://pixabay.com/users/mcmurryjulie/">mcmurryjulie</a> / Pixabay
An Overview of Bardet-Biedl Syndrome
At Patient Worthy, we are dedicated to the covering the latest stories about rare diseases that appear in the news cycle. While this is an important part of our mission…
<a href="https://pixabay.com/users/Parentingupstream/">Parentingupstream</a> / Pixabay
Clinical Trial Results With Velcade in Mantle Cell Lymphoma Inspire Continued Research
According to a story from curetoday.com, a recent Phase 2 trial has demonstrated that bortezomib (marketed as Velcade) has the potential to be an effective therapy for patients with mantle…
<a href="https://pixabay.com/users/JeongGuHyeok/">JeongGuHyeok</a> / Pixabay
Drug Fails to Make a Difference in Nonalcoholic Steatohepatitis
According to a story from Proactive Investors, the biotechnology company Conatus Pharmaceuticals recently reported that the company's drug Emricasan failed to achieve the primary endpoint in a Phase 2b clinical…
<a href="https://pixabay.com/users/skeeze/">skeeze</a> / Pixabay
A Girl With CDLK5 Deficiency Faces Spinal Surgery
According to a story from the Greenock Telegraph, twelve year old Megan McGarva, who was born with CDLK5 deficiency, will soon have to face a serious spinal operation. She has…
New Phase 1b/2a Trial for Pulmonary Sarcoidosis is on its Way!
Sarcoidosis is an inflammatory condition which can affect any organ in people of any age. The prognosis ranges from benign to debilitating. Granulomas, or clumps of cells, build up in…
