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We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. This holds the potential to cure geneticdiseases that have plagued families for generations.
The FDA cleared Welireg, which Merck picked up in its $1 billion takeover of Peloton Therapeutics, to treat certain tumors associated with a rare geneticdisease.
Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the geneticdisease.
Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.
The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare GeneticDiseases appeared first on Pharma Mirror Magazine. Catalent will further support process optimization and look to reduce material.
CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens.
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare geneticdiseases through the use of personalized antisense oligonucleotides (ASOs).
A team of researchers at the University of Georgia has identified the source of several symptoms in patients suffering from familial dysautonomia (FD), a rare and debilitating geneticdisease that primarily affects children.
Around 5,500 people with severe developmental disorders now know the genetic cause of their condition, thanks to a major nationwide study in the U.K. that will help improve diagnosis across the world.
Welireg, which Merck got through its purchase of Peloton Therapeutics, can now be used to treat an advanced form of kidney cancer, adding to an earlier clearance in a rare geneticdisease.
This week, researchers presented findings in how ancestry affects cancer treatment, nanotechnology and geneticdiseases. Here's a look at that and more.
Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.
Curing debilitating geneticdiseases is one of the great challenges of modern medicine. During the past decade, development of CRISPR technologies and advancements in genetics research brought new hope for patients and their families, although the safety of these new methods is still of significant concern.
At a far distant point in Earth’s ancient past, two separate, single-celled life forms — an archaeon and a bacteria — became one in an act either of symbiosis or enslavement, depending on which microbiologist you ask.
In any given cell at any given time, molecular processes are underway to seal cracks in the double helix or proofread the genetic code, all part of a perpetual upkeep program that maintains the body's status quo and staves off disease.
CF is a progressive geneticdisease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. Vertex remains committed to advancing CF research, with plans to expand the impact of their CFTR modulators and explore new genetic mutations.
While cancer is a geneticdisease, the genetic component is just one piece of the puzzle — and researchers need to consider environmental and metabolic factors as well, according to a research review by a leading expert at the University of Alberta.
In the world of rare geneticdiseases, exome and genome sequencing are two powerful tools used to make a diagnosis. A recent addition to the toolkit, RNA sequencing, has been demonstrated to help researchers narrow down disease candidate variants identified first on exome and genome sequencing.
There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).
Scientists may have solved the question of why Ashkenazi Jews are significantly more susceptible to a rare genetic disorder known as Gaucher disease—and the answer may help settle the debate about whether they are less susceptible to tuberculosis (TB).
After Orchard Therapeutics’ gene therapy for a rare geneticdisease was approved in March, Peter Marks, M.D., received a simple email from Commissioner Robert Califf, M.D. | After Orchard Therapeutics’ gene therapy for a rare geneticdisease was approved in March, Peter Marks, M.D.,
The Food and Drug Administration on Friday approved the first treatment for Rett syndrome, a geneticdisease mostly affecting girls that causes severe neurologic impairments, robbing them of the ability to communicate or control muscle movement. The new drug, called Daybue, is made by Acadia Pharmaceuticals.
On the heels of withdrawing Relyvrio from the U.S. and Canadian markets, Amylyx is now charting a path in Wolfram syndrome with promising interim Phase II data for its lead asset AMX0035.
Despite beating analyst expectations, Takeda sustains a mid-stage defeat in a rare genetic disorder, leaving the fate of an investigational enzyme replacement therapy uncertain.
CF is a rare, life-shortening geneticdisease that is estimated to impact over 88,000 people across the globe. The progressive multi-organ disease affects the patients’ lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.
million grant from The National Institutes of Health to develop methods that will improve genetic risk estimates – polygenic risk scores – for specific diseases in people from diverse populations and mixed ancestries. LOS ANGELES (June 16, 2021) – UCLA Health will receive a $4.8
Credit: Gretel Nicholson, EXRC Scientists have discovered a new geneticdisease, which causes some children’s brains to develop abnormally, resulting in delayed intellectual development and often early onset cataracts.
CAMP4’s RNA-based therapies focus on geneticdiseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder. Jupiter joins a wave of biotech IPOs this year, stepping into a competitive space while broadening treatment options for CNS patients.
Human populations have waxed and waned over the millennia, with some cultures exploding and migrating to new areas or new continents, others dropping to such low numbers that their genetic diversity plummeted. In some small populations, inbreeding causes once rare geneticdiseases to become common, despite their deleterious effects.
MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare geneticdiseases. The new deal excludes oncology and other therapeutic areas outside the scope of geneticdisease.
The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for GeneticDisease (iCGD) to advance life sciences.
The company’s olezarsen cleared a late-stage study, eliciting a sharp reduction in triglyceride levels in patients with familial chylomicronemia syndrome. Ionis plans to submit a New Drug Application to the FDA.
Prime Medicine has been given the go-ahead by the FDA for the first human trial of its prime editing technology, which promises to deliver one-shot therapies for a range of severe geneticdiseases.
Streamlining the regulatory process, the AMP BGTC will also facilitate cost-efficient vector production to increase access for patients with rare and ultra-rare geneticdiseases. The collaboration creates a reusable standardised method that minimises upfront costs and helps to reduce development barriers.
After scoring an FDA nod Monday for the first gene therapy to treat the rare geneticdisease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare geneticdisease metachromatic leukodystrophy in the U.S.,
Tessera Therapeutics chief scientist Michael Holmes had just finished presenting a much-anticipated peek at a technology the company had previously said could “ revolutionize genetic medicine ” and “cure nearly any geneticdisease.”
Scientists have developed a test that could greatly improve quality of life for infants with homocystinuria (HCU), a congenital disease that—if not treated early—causes serious complications. Research demonstrating the efficacy of this test was published today in Clinical Chemistry.
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