Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
In recent years, gene therapy has expanded to enormous heights. This experimental technique allows scientists to add, delete, edit, or otherwise use genes to address genetic malfunctions and disorders. Now,…
According to Charcot-Marie-Tooth News, biotechnology company Helixmith recently began a Phase 1/2a clinical trial to test the efficacy of Engensis (VM202) for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). The…
Last Tuesday, the FDA rejected BioMarin Pharmaceutical's gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment…
Today, American Gene Technologies (AGT) announced that they will soon be beginning a Phase 1 clinical trial for AGT103-T, a potential treatment for HIV. The unique gene therapy, developed…
According to a story from the CureDuchenne Blog, Dr. Kevin Flanigan of Nationwide Children's Hospital has recently dosed two Duchenne muscular dystrophy patients with an experimental gene therapy. This gene…
uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington's disease. Two patients are included so far, with one being given…
According to Medical Xpress, a new gene therapy has been studied for the treatment of Duchenne muscular dystrophy. This treatment, SRP-9001, has been shown to deliver micro-dystrophin and improve functional…
In a recent press release, biotechnology company Sarepta Therapeutics announced positive findings from their clinical trial studying SRP-9003 as a treatment for limb-girdle muscular dystrophy (LGMD) type 2E. This…
According to a story from BioPharma Dive, the drug company Sarepta Therapeutics is pushing forward with its plans to develop a gene therapy for limb-girdle muscular dystrophy (LGMD). The company…
According to a story from interestingengineering.com, the capabilities of CRIPSR gene editing technology have been creating a buzz in the medical field for years now, with many seeing the technology…
By Lauren Thayer from In The Cloud Copy Hemophilia is a medical condition in which a person’s blood clotting ability is severely reduced. This inability to properly clot leads to…
A recent BioSpace press release shares some exciting news for patients with non-small cell lung cancer, medullary thyroid cancer, and other thyroid cancers caused by RET gene mutations. Retevmo (selpercatinib) is the…
For those in the rare disease community, you know that it can take a long time to reach a diagnosis for your condition: an average of 4.8 years! But upon diagnosis, many…
The last decades have seen significant progress when it comes to medical care for rare diseases. Tasks doctors once slaved over were transformed with better speed and efficiency by modern…
As reported in Biospace, Adverum Biotechnologies, a gene therapy company focused on clinical stage trials for rare and ocular diseases with unmet needs, has successfully dosed a patient in their…
According to Batten Disease News, Invitae and Axovant Gene Therapies partnered up to offer free genetic testing in North America. This will focus on children who are thought to…
In a post on the Metachromatic Leukodystrophy (MLD) Foundation's website, the organization discusses the possibilities of using gene therapy as a treatment for this rare genetic disorder. The page discusses…
As reported in Biospace, a new gene therapy has made notable progress towards curing x-linked retinitis pigmentosa, a disease responsible for sight loss in men. Until now, the disease irreversibly…
According to Microbiome Times, Ilya Pharma has received a BIO-X® grant to study gene therapy for patients with inflammatory bowel disease. Their chemokine-based gene therapy drug candidates seek to…
Research findings can sometimes come from unexpected places. For example, according to Parkinson's News Today, the benefits and shortcomings of an investigative gene therapy were explored through the post-mortem analysis…
Cystic fibrosis is caused by a mutation in the gene CFTR. This gene involves a protein that affects the chloride on cells; without this chloride the surface of the…
According to a story from the National Gaucher Foundation, the use of gene therapy in order to treat Gaucher disease has long been floated in medical theory, but only in…
During the 1990s, researchers had a dream that by 2020, every individual would have access to their own genome. This would aid in their diagnosis and treatment as soon as…
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The gene therapy Zolgensma recently won approval last summer in 2019 to be used for infants who have spinal muscular atrophy. The company AveXis, the makers of the Zolgensma gene…
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