XTalks

AUGUST 6, 2020

Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant. Epidiolex is a plant-derived CBD solution developed by GW Pharmaceuticals. TSC is a rare genetic disease that affects approximately 1 in 6,000 people. Symptoms can range from being mild to more severe or life-threatening.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 103

Genome Genomics Genetics Genome Project 103

XTalks

NOVEMBER 10, 2023

Acute manifestations include thrombocytopenia, microangiopathic hemolytic anemia, bleeding episodes, headache and abdominal pain, while chronic ones include stroke, cardiovascular disease and damage to organs. As a rare disease, cTTP affects less than 1,000 individuals in the US.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 80

Genome Genomics Genetics Genome Project 80

The Pharma Data

DECEMBER 17, 2020

About Lysogene Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 52

XTalks

APRIL 15, 2021

Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. The reverse could also be true whereby the development of coronary artery disease may impact heart rate. HFrEF vs. HFpEF.

Development 98

Development Drugs Genome Genomics 98

pharmaphorum

JANUARY 27, 2023

Late last year, pharmaphorum caught up with Dr Karen Mullen, chief medical officer and VP of clinical & medical affairs at global drug development consultancy Boyds. We talk a lot in clinical trials and drug development about benefit,” Dr Mullen said.

Clinical Trials 111

Clinical Trials Clinical Trials Trials Development 111

The Pharma Data

DECEMBER 27, 2020

Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. Sarepta Therapeutics, Inc.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials In-Vivo 52

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

Genome 98

Genome Genomics Medicine Genetic Disease 98

Delveinsight

AUGUST 17, 2021

Food and Drug Administration, which could have entered the ultra-rare genetic disease market. The rarity of disease – 1.36 Ipsen takes back its Application for New Drug Palovarotene that could be used to treat new bone formation in people with fibrodysplasia ossificans progressive. person/million around the globe.

FDA Approval 98

FDA Approval Medicine Vaccination Vaccine 98

The Pharma Data

NOVEMBER 11, 2020

Nasdaq: INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the third quarter ended September 30, 2020 and provided recent business highlights.

Clinical Trials 40

Clinical Trials Clinical Trials Trials Development 40

The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Shortening of telomeres is associated with the symptoms of aging, heart disease, DNA damage and uncontrolled cell replication, which can lead to cancer.

DNA 52

DNA Scientist Genetic Disease Research 52

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

NOVEMBER 2, 2020

President Worldwide Research and Development at BioMarin. “Our extensive development program has been focused on addressing the underlying cause of achondroplasia. BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases.

Drugs 40

Drugs Containment Trials Marketing 40

The Pharma Data

AUGUST 15, 2021

The WELIREG label contains a boxed warning that exposure to WELIREG during pregnancy can cause embryo-fetal harm. About Von Hippel-Lindau Disease. This is a rare genetic disease with an estimated incidence of 10,000 people in the U.S. As an inhibitor of HIF-2?, WELIREG reduces transcription and expression of HIF-2?

Hormones 52

Hormones Clinical Trials Clinical Trials Trials 52

The Pharma Data

DECEMBER 3, 2020

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Roche’s Chief Medical Officer and Head of Global Product Development.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR. It’s a great tool for controlling gene expression.”.

DNA 52

DNA Gene Gene Silencing Genome 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

AUGUST 3, 2021

The use of a sham procedure in STEER is included to provide a comparison group for an unbiased collection and assessment of efficacy, safety and tolerability of OAV-101 IT for this older population where the disease progression is slower. Spinal Muscular Atrophy (SMA) ? Source link: [link] /.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

JANUARY 25, 2021

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

Sales 52

Sales Marketing Containment Development 52

The Pharma Data

OCTOBER 28, 2020

NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics.

Protein 40

Protein Production Development Drugs 40

The Pharma Data

JANUARY 9, 2021

President of Worldwide Research and Development at BioMarin. No participants developed inhibitors to Factor VIII, or thromboembolic events. No participants developed inhibitors to Factor VIII, and no participants withdrew from the study. No participants have developed thrombotic events. About BioMarin.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52