Posts by: Marisa Wexler MS

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PT001, an investigational treatment for pulmonary arterial hypertension (PAH). The FDA gives this designation to therapies with the ... Read more

June 21, 2021June 21, 2021

New Strategy Corrects CF Splicing Defects in Lab Models

Cystic Fibrosis News Today

A new strategy to correct so-called splicing mutations could help deliver nucleic acid therapies called oligonucleotides to cells in the lung, a new study suggests. This approach may aid in ... Read more

June 21, 2021June 21, 2021

Oral Aubagio Approved in EU for Children With RRMS, Ages 10-17

Multiple Sclerosis News Today

The European Commission (EC) has approved Aubagio (teriflunomide) for the treatment of children and adolescents, ages 10 to 17, with relapsing-remitting multiple sclerosis (RRMS). Aubagio, approved for adults with RRMS since ... Read more

June 21, 2021June 21, 2021

Results: Ponvory Outperforms Aubagio in OPTIMUM Clinical Trial

Multiple Sclerosis News Today

In the OPTIMUM clinical trial, Ponvory (ponesimod) significantly outperformed Aubagio (teriflunomide) in reducing relapse rates, fatigue, and evidence of brain damage among people with relapsing multiple sclerosis (MS). Based on these ... Read more

June 17, 2021June 17, 2021

CFF, Deep Science Ventures Team Up to Advance Gene Therapies

Cystic Fibrosis News Today

A collaboration between the Cystic Fibrosis Foundation and Deep Science Ventures aims to identify technologies that might overcome the challenges inherent in developing gene therapies for cystic fibrosis (CF). The effort is ... Read more

June 16, 2021June 16, 2021

FKBP5 Protein May Be Cortisol Activity Biomarker

Cushing's Disease News

Measuring the levels of the RNA molecule that provides instructions to make a protein called FKBP5 could be useful for assessing cortisol activity in people with Cushing’s disease, a new ... Read more

June 16, 2021June 16, 2021

Medicaid Spending on Hemophilia Therapies in US Tripled in 2005-19

Hemophilia News Today

Spending on treatments for hemophilia by Medicaid, a U.S. government health insurance program for select groups, more than tripled from 2005 to 2019, an analysis reported. Its researchers expect this finding ... Read more

June 16, 2021June 17, 2021

Data on T-cell Repertoire May Help in Understanding MS Development

Multiple Sclerosis News Today

T-cells in the fluid around the brain share a large percentage of receptors among different multiple sclerosis (MS) patients, a new report suggests. Better understanding the diversity of T-cell receptors ... Read more

June 16, 2021June 16, 2021

Abstract on How Avacopan Affects Kidney Function Makes Top 10 List

ANCA Vasculitis News

Treatment with ChemoCentryx’s investigational therapy avacopan can improve kidney function in people with ANCA-associated vasculitis to a greater extent than steroids, especially among individuals with advanced kidney disease, clinical trial ... Read more

June 16, 2021June 16, 2021

BiomX’s Phage Cocktail Seen to Kill Resistant P. aeruginosa in Studies

Cystic Fibrosis News Today

BX004, a phage therapy candidate for Pseudomonas aeruginosa infections in people with cystic fibrosis (CF), was able to effectively kill a range of antibiotic-resistant bacterial strains under laboratory conditions, new data show. These findings ... Read more

June 15, 2021June 15, 2021

First People Dosed With Investigational Therapy EDT001

Cystic Fibrosis News Today

The first participants have been dosed in the first clinical trial testing EDT001, an investigational therapy for cystic fibrosis (CF) being developed by Enterprise Therapeutics. The trial is being conducted ... Read more

June 15, 2021June 15, 2021

FDA Rejects Expansion of Aubagio for Pediatric MS

Multiple Sclerosis News Today

The U.S. Food and Drug Administration (FDA) has rejected an application requesting the expansion of Aubagio (teriflunomide) for the treatment of children and adolescents, ages 10 to 17, with relapsing ... Read more

June 14, 2021June 14, 2021

$5M Grant Supports Phase 1 Trial of AAV2-BDNF, Potential Gene Therapy

Alzheimer's News Today

The National Institute on Aging (NIA) has awarded a five-year grant worth up to $5 million to support a clinical trial that will test AAV2-BDNF, an investigational gene therapy, in ... Read more

June 14, 2021June 14, 2021

Amyl Raises Funds to Develop Therapies for Amyloidosis

FAP News Today

Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial amyloid polyneuropathy (FAP) and other forms of ... Read more

June 14, 2021June 14, 2021

Acer Plans Pivotal Trial of Edsivo in COL3A1-positive vEDS

Ehlers-Danlos News

Following a meeting with the U.S. Food and Drug Administration (FDA), Acer Therapeutics is planning to launch a potentially pivotal clinical trial to evaluate the safety and efficacy of its ... Read more