Posts by: Marta Figueiredo PhD

FAP News Today

The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation RNA interference (RNAi) therapy candidate for familial amyloid polyneuropathy (FAP). Hereditary ATTR ... Read more

September 20, 2021September 21, 2021

MS Patients in UK Now Offered COVID-19 Booster Shots

COVID-19 News, Multiple Sclerosis News Today

The U.K.’s National Health Service (NHS) has started its COVID-19 vaccine booster program, offering a third shot — preferably with the Pfizer-BioNTech vaccine — to at-risk populations, including people with ... Read more

September 20, 2021September 20, 2021

Out of ‘Stealth’ Mode, QurAlis Expects First QRL-201 Trial in 2022

ALS News Today

QurAlis is advancing the development of QRL-201, its “groundbreaking” lead treatment candidate for amyotrophic lateral sclerosis (ALS), and expects to initiate its first clinical trial in late 2022. The stem ... Read more

September 20, 2021September 20, 2021

Review Study Does Not Recommend Adding Antibiotics to Standard IPF Care

Pulmonary Fibrosis News

Adding co-trimoxazole or doxycycline — two broad-spectrum antibiotics — to standard care does not significantly slow lung function decline or prolong survival in people with idiopathic pulmonary fibrosis (IPF), a ... Read more

September 17, 2021September 17, 2021

ATYR1923 Found Safe, Effective for Pulmonary Sarcoidosis Patients

COVID-19 News, Sarcoidosis News

aTyr Pharma’s experimental therapy ATYR1923 safely and effectively improves lung function and reduces symptoms in adults with pulmonary sarcoidosis, while lowering the need for oral corticosteroids, top-line data from a ... Read more

September 16, 2021September 16, 2021

Phase 1b Trial of Oral DNL343 in ALS Patients Begins Dosing

ALS News Today

A Phase 1b clinical trial evaluating the safety and pharmacological profile of DNL343, Denali Therapeutics’ investigational oral therapy, in adults with amyotrophic lateral sclerosis (ALS) has started dosing participants. The ... Read more

September 16, 2021September 16, 2021

Ferriprox Approved in Brazil to Treat Iron Overload in SCD Patients

Sickle Cell Disease News

Regulators in Brazil have approved Ferriprox (deferiprone), Chiesi Global Rare Diseases’ iron-binding oral treatment, for blood transfusion-induced iron overload in people with sickle cell disease (SCD) or other anemias. Ferriprox’s use ... Read more

September 15, 2021September 15, 2021

New ATP1A1 Mutation Identified in Adolescent with Intermediate CMT

Charcot-Marie-Tooth News

A newly identified mutation in the ATP1A1 gene associated with an intermediate form of Charcot-Marie-Tooth disease (CMT) was found in an adolescent in Spain, scientists report. The mutation, detected through ... Read more

September 15, 2021September 15, 2021

Pandemic Placed Socioeconomic Hardships on CF Patients

COVID-19 News, Cystic Fibrosis News Today

The COVID‐19 pandemic increased socioeconomic instability among people with cystic fibrosis (CF), mostly in terms of employment and ability to afford food, according to data from a single-center U.S. study. Notably, patients ... Read more

September 15, 2021September 15, 2021

Amylyx Plans to File for Approval of AMX0035 in US

ALS News Today

Amylyx Pharmaceuticals plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in the coming months for approval of its investigational oral therapy AMX0035 ... Read more

September 15, 2021September 15, 2021

Blood NfL Levels May Mark SMA Severity, Therapy Efficacy in Very Young

SMA News Today

Blood levels of neurofilament light chain (NfL) — a marker of nerve cell damage — in children and adolescents with spinal muscular atrophy (SMA) are significantly higher than those of ... Read more

September 15, 2021September 15, 2021

Be Proactive in Managing Bladder, Bowel Problems, Nurses Advise

Multiple Sclerosis News Today

Bladder and bowel problems, which affect more than half of multiple sclerosis (MS) patients, should be assessed and monitored regularly to better adapt treatment plans and ultimately improve patients’ quality of ... Read more

September 14, 2021September 14, 2021

PWS Community Asked to Take Part in Nov. 4 FDA Virtual Meeting on LV-001

Prader-Willi News

The U.S. Food and Drug Administration (FDA) is holding a public advisory committee meeting regarding LV-101 (intranasal carbetocin) as a potential treatment of distress behaviors and insatiable hunger in Prader-Willi ... Read more

September 14, 2021September 15, 2021

Omega-3 Supplements May Slow Memory Decline

Alzheimer's News Today

Six months of daily supplements of omega-3 increased the levels of two biomarkers of inflammation and nerve damage in the cerebrospinal fluid (CSF) of people with Alzheimer’s disease, according to ... Read more

September 14, 2021September 14, 2021

Abnormal DNA Repair Mechanism May Be Therapeutic Target

Huntington's Disease News

The interaction between FAN1 and MLH1 — two DNA repair proteins known to be genetic modifiers of Huntington’s disease — protects against further expansion of disease-causing CAG repeats, according to ... Read more