The parents had brought their baby girl home only four days ago when they received an urgent call from the hospital asking them to bring the baby right back for…
One of the most successful healthcare services benefiting the public is newborn screening. Unfortunately, this life-saving health initiative is not provided for all rare diseases in some U.S. states. As…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
When Eric and Samantha Rovinetti learned that they were pregnant with their first child, they were overjoyed. They could not wait to welcome baby Adrianna into the world. But shortly…
In 2022, the 25th Annual Meeting of the American Society of Gene & Cell Therapy took place from May 16th-19th. During the meeting, researchers and other stakeholders discussed the latest…
It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary…
According to a July 13 news release, biopharmaceutical company Kedrion Biopharma ("Kedrion") recently treated its first pediatric patient within the Phase 3 KIDCARES10 clinical trial. Within the trial, Kedrion…
Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…
There are multiple gene mutations associated with severe combined immunodeficiency (SCID), a rare genetic disorder which severely inhibits - or stops - immune function. But according to New Atlas, an…
Severe Combined Immunodeficiency Severe combined immunodeficiency (SCID) is a group of diseases caused by mutations that affect how the immune system works to fight infections. These anomalies are usually passed…
While gene therapy offers the promise of addressing and treating many genetic disorders, creating a better quality of life for many people, there are also ethical issues associated with…
According to a story from BioSpace, SIRION Biotech GmbH and Mustang Bio, Inc. have announced a new agreement for licensing the use of SIRION's proprietary LentiBOOST™ technology for use by…
Michael and Armené Kapamajian were at home in Los Angeles settling in with their one week old baby, Sasoun, when their doctor called and asked them to come in…
Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…
The Bettuchi's Amanda Bettuchi and her husband have 3 children. Their middle child, Shane, was born with severe combined immunodeficiency (SCID). Essentially, this means he was born with practically no…
According to a story from Express Digest, four year old Khloe Land of Oregon stepped up to the plate to help her brother get a critical treatment that could very…
According to a press release from American biotechnology company Fortress Biotech, the American Food and Drug Administration (FDA) recently awarded Regenerative Medicine Advanced Therapy designation to the experimental gene therapy…
ABC News and Good Morning America brightened up our morning by featuring a story of 1-year-old Alle Jilg, whose life was saved by total stranger's bone marrow donation. Alle is…
SCID Severe combined immunodeficiency (SCID) is a rare disease which causes patients to have little to no immune response. With practically no immune system, SCID patients are very susceptible to…
Researchers at the University of Colorado Anschutz Medical Campus have uncovered a never-before discovered immunodeficiency disease. How It Came About It started with a newborn boy who had a myriad…
We just read exciting news for the SCID community from Stanford researchers. The original study was published in Nature Communications, but a summary appeared in Medical Xpress. The researchers have…
Severe Combined Immunodeficiency Severe Combined Immunodeficiency (SCID) is a rare disease which causes patients to practically have no immune system at all. That means that for these patients, even a…
ADA-SCID ADA-SCID is a form of Severe Combined Immunodeficiency (SCID) which is caused by Adenosine Deaminase Deficiency. A lack of the ADA gene in the body results in impaired development of the…
A frequent impediment to rare disease research is cost. It's unfortunate, but it's the reality of the world we live in. That means every fundraising campaign and every dollar raised…
According to a report by BioSpace, a new treatment for SCID could be around the corner. Mustang Bio, Inc. (Mustang), and St. Jude Children’s Research Hospital recently announced a partnership…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
