Hannah Creel was only 18 months old when she was first diagnosed with cystinosis, a rare disorder that affects all organs in the body, in particular the kidneys. Her parents…
WLNS Brooklyn Michigan is sharing news about Jaxon Meschke, a four-year-old boy with a rare disease called cystinosis. Jaxon’s body has been fighting the disease since he was six…
A new study is in the works, and it will investigate how cystinosis, a genetic disorder characterized by an accumulation of the amino acid cystine in the cells, leads to…
According to a story from Market Watch, the gene therapy company AVROBIO, Inc. has recently announced that its investigational gene therapy candidate AVR-RD-04 has earned Orphan Drug designation from the…
At the beginning of March 2021, gene therapy company AVROBIO, Inc. shared that its investigational gene therapy, AVR-RD-04, was given Orphan Drug designation by the European Commission (EC). This treatment…
According to a story from ktvl.com, seven year old Emma Suetta, of Etna, California lives with a rare disease called cystinosis, and she is doing her part to help find…
By Rebekah Palmer The rare disease community has an all too common problem of not using precise language when it comes to addressing the people and the needs of…
Written by Rebekah Palmer We have passed the 30th anniversary of the Americans with Disabilities Act. The landmark legislation that prohibits discrimination of disabled people in public spaces is rarely…
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…
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…
My name is Mika Jayne Covington. I’m 29 years old and I live with an ultra-rare disease called cystinosis. I am also a transgender woman and my pronouns are she,…
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…
Because I will be looking for a job from a rare but certainly not unique place of being, I decided to reach out to a mentor who lives with the…
AVROBIO has recently received the Orphan Drug designation for AVR-RD-04, a gene therapy for the treatment of cystinosis. This treatment takes the stem cells of those affected and modifies them…
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,…
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…
Note: The author does hold immense compassion for children and teens and their families living with rare disease. Compassion is empathetic in sorrow, rather than wishing there is no sorrow,…
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…
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…
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…
According to a story from kdrv.com, cystinosis is a very rare disease that only affects about 500 people in the US. Two of those patients are Emma Suetta and Everly…
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…
May is Cystinosis Awareness Month! During the month (and all year long!) we strive to bring together the cystinosis patient community and their families -- and the Cystinosis Research Network's…
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…
There was a time when most children diagnosed with a rare condition did not live until adulthood. But now, with advances in medicine, many individuals are living long, full lives…
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