As bluebird bio ("bluebird") waited on FDA feedback regarding the manufacturing process for lovotibeglogene autotemcel (lovo-cel) genetherapy, the company disclosed that it would most likely miss its Q1 submission…
A therapy is granted Orphan Drug designation if the FDA believes that this therapy will treat, prevent, or diagnose rare conditions. Rare conditions, in the United States, are those affecting…
Children all across the globe enrolled in a study to evaluate Upstaza, a gene therapy medication, for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. As part of their…
Medical research is crucial in better understanding diseases and discovering novel treatment options. Unfortunately, there is often one large barrier to research: funding. This is especially true within the rare…
The National Institutes of Health (NIH) held its in-person Rare Disease Day at NIH convergence on February 28, 2023. This was the first time in a while that the event…
In November 2022, the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) approved Upstaza (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The product, which…
According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics,…
According to a story from Globe Newswire, the gene therapy company uniQure recently announced that it has received conditional marketing authorization for its gene therapy etranacogene dezaparvovec (marketed as HEMGENIX®).…
Dr. Muna Naash received her PhD from Baylor College of Medicine, where she studied retinal cell and molecular biology. Throughout her career, Dr. Naash has become known for her work…
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Growing up, Priscilla Veneklause watched as her father struggled to manage his rare genetic disorder: adrenoleukodystrophy (ALD). Unfortunately, her father lost his fight with ALD when Priscilla was still…
Currently, the standard-of-care for individuals living with hemophilia B is intravenous factor IX infusions (prophylaxis). These infusions can be time-consuming, require adherence to a strict schedule, and may not…
According to a late October 2022 news release from gene therapy company AVROBIO, the sixth patient has been dosed in a Phase 1/2 clinical study evaluating AVR-RD-04 for patients…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
As discussed in Part I of this article, a recent HDBUZZ publication featured a news item stating that due to several severe adverse events occurring during the initial AMT-130 trial…
When we ‘do the math’ it adds up to the novel AMT-130 gene therapy receiving positive results from uniQure’s clinical trial investigating a small group (N=4) of individuals who were…
On August 25th, 2022, the American Society of Gene & Cell Therapy held a webinar as part of its Lunch and Learn series, which is intended to help people living…
Both the 3rd Annual Gene Therapy for Neurological Disorders meeting and the ADVANCE 2022 Sanfilippo Community Conference were held in July 2022. During both meetings, researchers presented new data from…
Before you read on, make sure you take a look at Pt. 1 of our interview, which goes over what PGAP3 is, Lucy's story, and the reasons behind why Zach and Geri…
Zach and Geri Landman are devoted parents and physicians – a pain specialist and pediatrician, respectively – committed to making the world a better place for those around them. They…
In normal development, genes are usually inherited in pairs - with one copy from the father and one inherited from the mother. When one copy of the TCF4 gene, which…
The FDA has just announced that they have lifted the temporary hold placed on pheNIX gene therapy. This gene therapy was created by Homology Medicine as an investigational treatment for…
Pancreatic cancer is one of the deadliest forms of cancer. According to the American Cancer Society (ACS), only about 11% of patients are expected to survive more than five years…
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…
CureDuchenne is a nonprofit whose mission is to find a cure for Duchenne muscular dystrophy (DMD), a rare progressive disease characterized by muscular degeneration. It is diagnosed in one individual…
PTC Therapeutics Inc. has just announced that their gene therapy called Upstaza has received a positive opinion from the EMA. After the therapy is ratified by the European Commission (EC),…
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