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…
Near the start of August 2022, biotechnology company Inmagene Biopharmaceuticals ("Inmagene") and biopharmaceutical company HUTCHMED shared via news release that the first participant had been dosed in a Phase…
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…
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…
Armené and Michael Kapamajian live in LA. Their son Sasoun was diagnosed with severe combined immunodeficiency (SCID). This means that his immune system is compromised and he is extremely vulnerable…
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…
According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…
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Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…
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 story from the Warwick Beacon, the first ever primary immunodeficiency (PI) awareness and fundraising walk in Rhode Island's history was held recently at Warwick City Park. The…
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…
According to a story from Australasian Science Magazine, the dawn of the gene therapy "revolution" is upon us. While there are only a handful of gene therapies that have actually…
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…
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…
About Graft-Versus-Host-Disease (GvHD) Graft-Versus-Host-Disease (GvHD) occurs when a patient's body rejects cells which have been transplanted. For instance, it may develop following a stem cell transplant or a bone marrow transplant. The…
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