Watch “Walk In My Shoes” And Learn How Chandler Keeps Smiling With Cystinosis
Free-Photos / Pixabay

Watch “Walk In My Shoes” And Learn How Chandler Keeps Smiling With Cystinosis

The documentary "Walk in My Shoes” takes viewers through a lifetime with cystinosis with Chandler and his family. After Chandler was diagnosed with the rare disease as an infant, making…

Continue Reading Watch “Walk In My Shoes” And Learn How Chandler Keeps Smiling With Cystinosis
The Brighter Days Care Package Program for Cystinosis Patients and Families has Launched
source: pixabay.com

The Brighter Days Care Package Program for Cystinosis Patients and Families has Launched

The Cystinosis Research Network (CRN) has created a new program to bring light to cystinosis families during these uncertain times. It is called the Brighter Days Care Package Program. Brighter…

Continue Reading The Brighter Days Care Package Program for Cystinosis Patients and Families has Launched
Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis
source: pixabay.com

Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis

As reported in Business Fortnight, the top level gene therapy company AVROBIO has announced successful clinical trials for investigational drugs for Fabry disease and cystinosis. The CEO of AVROBIO, Geoff…

Continue Reading Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis
Study Examines Neurological Differences in Cystinosis Patients
source: pixabay.com

Study Examines Neurological Differences in Cystinosis Patients

In a study published recently in the Orphanet Journal of Rare Diseases, a team of researchers identified distinct variations in the neuroanatomical and neuropsychiatric phenotypes of cystinosis patients. Phenotype refers to visible,…

Continue Reading Study Examines Neurological Differences in Cystinosis Patients
AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program
source: pixabay.com

AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program

According to a story from Financial Buzz, the gene therapy company AVROBIO recently presented updated data on February 10th, 2020 at the 16th Annual WORLDSymposium being held in Orlando, FL. These updates…

Continue Reading AVROBIO Provides Updates on its Fabry Disease Program and Cystinosis Program
First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis
source: pixabay.com

First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis

According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…

Continue Reading First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis

A Cystinosis Story: No Patient Should Have to Fight for Access to Their Medication

Cystinosis Cystinosis is a rare disease that causes cystine, an amino acid, to build up in the body's organs. However, there is treatment that can prevent this accumulation. Normally, patients…

Continue Reading A Cystinosis Story: No Patient Should Have to Fight for Access to Their Medication
CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients
Skitterphoto / Pixabay

CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients

According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…

Continue Reading CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients
Rare Patients, Rare Miracles: Siblings With Cystinosis Get Transplanted Kidneys from the Same Donor
skeeze / Pixabay

Rare Patients, Rare Miracles: Siblings With Cystinosis Get Transplanted Kidneys from the Same Donor

According to a story from KUT News, Ava Shepperd and John Ben Shepperd, aged 14 and 18 respectively, always knew that they would eventually need to get kidney transplants. This…

Continue Reading Rare Patients, Rare Miracles: Siblings With Cystinosis Get Transplanted Kidneys from the Same Donor

A Regulatory Agency in Canada is Calling Down the Thunder on Rare Disease Drug Prices

According to a story from Yahoo News, the Patented Medicine Prices Review Board (PMPRB), a small agency of the Canadian government that employs less than 100 people in total, is…

Continue Reading A Regulatory Agency in Canada is Calling Down the Thunder on Rare Disease Drug Prices

Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa

The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…

Continue Reading Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa
Kidney Disease Drug May Have Future Treating Mitochondrial Disorders
argzombies / Pixabay

Kidney Disease Drug May Have Future Treating Mitochondrial Disorders

A publication at Science Daily recently announced that findings from Children's Hospital of Philadelphia suggested that cysteamine bitartrate, already used for nephropathic cystinosis (a rare kidney disease), might potentially benefit patients…

Continue Reading Kidney Disease Drug May Have Future Treating Mitochondrial Disorders
Close Menu