Posts by: Marisa Wexler MS

Patients with idiopathic pulmonary fibrosis (IPF) with chronic cough who are being treated with the investigational oral medication NP-120 (ifenprodil) in a small clinical trial tended to experience a reduction ... Read more

January 14, 2022January 14, 2022

Blocking JNK Signaling Pathway May Be Useful for Treating Rett

Rett Syndrome News

A cellular signaling pathway called the JNK pathway is overactive in cell and animal models of Rett syndrome, and blocking it lessens signs of the disease in these models, according ... Read more

January 14, 2022January 17, 2022

Avrobio Stopping Its Gene Therapy Program for Fabry Disease

Fabry Disease News

Note: This story was updated Jan. 17, 2022, to note that Avrobio is deprioritizing its Fabry program. Avrobio is stopping its gene therapy program for Fabry disease in favor ... Read more

January 14, 2022January 14, 2022

AVR-RD-01, Fabry Gene Therapy, Continuing to Show Safety in Trials

Fabry Disease News

AVR-RD-01, an experimental gene therapy for Fabry disease, is continuing to show a positive safety profile in its clinical trials, with no serious adverse events related to the treatment reported, ... Read more

January 14, 2022January 14, 2022

Cudetaxestat Well-tolerated in Esbriet, Ofev Combos in Phase 1 Trial

Pulmonary Fibrosis News

Cudetaxestat (BLD-0409), an experimental medication for idiopathic pulmonary fibrosis (IPF) from Blade Therapeutics, was well-tolerated among healthy volunteers when given in combination with other IPF treatments — specifically nintedanib and ... Read more

January 14, 2022January 14, 2022

New Capsule Form of Adrulipase Alfa for EPI May Soon Enter Trial

Cystic Fibrosis News Today

First Wave BioPharma has filed two new patents related to adrulipase, a yeast-derived enzyme that the company is developing to treat exocrine pancreatic insufficiency in people with cystic fibrosis (CF). One ... Read more

January 14, 2022January 14, 2022

PANTHERx Rare Pharmacy to Distribute Recorlev Therapy in US

Cushing's Disease News

Xeris Pharmaceuticals has selected the specialty pharmacy PANTHERx Rare to distribute Recorlev (levoketoconazole), the company’s therapy that was recently approved to treat adults with endogenous Cushing’s syndrome in whom surgery is ... Read more

January 13, 2022January 13, 2022

First Zebrafish Model of Sanfilippo Offers New Research Opportunities

Sanfilippo News

In a world-first, a team of scientists in Australia has developed a model of Sanfilippo syndrome type A in zebrafish. “By adding a zebrafish model to the toolbox for Sanfilippo ... Read more

January 13, 2022January 13, 2022

Abnormal Brain Activity Found in PWS Patients When They See Food

Prader-Willi News

In people with Prader-Willi syndrome (PWS), certain parts of the brain associated with decision-making and reward processing are less active in response to the sight of food, a study has ... Read more

January 13, 2022January 13, 2022

Proposal That Medicare Covers Aduhelm Only for Trials Sparks Outrage

Alzheimer's News Today

A new proposal in the U.S. that Medicare cover the cost of Aduhelm (aducanumab) only for people enrolled in qualifying clinical trials has been met with outrage from the Alzheimer’s ... Read more

January 13, 2022January 13, 2022

CF Foundation Awards $3.5M to Develop Antibiotic ACG-701

Cystic Fibrosis News Today

The Cystic Fibrosis Foundation has awarded up to $3.5 million to Aceragen to support the clinical development of ACG-701, an investigational antibiotic to treat pulmonary exacerbations in people with cystic ... Read more

January 13, 2022January 13, 2022

Oral Edaravone Under FDA Priority Review, Decision Expected in May

ALS News Today

A decision is expected by May from the U.S. Food and Drug Administration (FDA) on an application seeking approval of an oral formulation of edaravone (MT-1186) — one with a ... Read more

January 13, 2022January 13, 2022

PRO Tool May Better Address MG Symptoms Most Affecting Patients

Myasthenia Gravis News

By analyzing available research and conducting nearly 100 interviews with patients, an international team of researchers crafted a new tool to measure the severity of myasthenia gravis (MG) based on ... Read more

January 12, 2022January 12, 2022

Kaftrio Approved for Children Ages 6 and Up in European Union, UK

Cystic Fibrosis News Today

The European Commission has expanded its approval of Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), to treat children with cystic fibrosis (CF) as young as 6 who have the most common type of ... Read more

January 12, 2022January 12, 2022

Enrollment Complete for Phase 2 Trial Testing Anti-fibrotic PLN-74809

Pulmonary Fibrosis News

Enrollment has been completed for INTEGRIS-IPF, a Phase 2a trial testing PLN-74809, an experimental oral anti-fibrotic therapy, in people with idiopathic pulmonary fibrosis (IPF). Pliant Therapeutics, the company developing PLN-74809 ... Read more