Posts by: Marta Figueiredo PhD

Marta Figueiredo PhD

January 17, 2022January 17, 2022

Despite Taking Spinraza, SMA Type 1 Children Show Nonmotor Problems

SMA News Today

Despite its well-reported benefits in motor function, Spinraza (nusinersen) may not be as effective at preventing or easing nonmotor complications, such as facial deformities, feeding problems, and scoliosis, in children ... Read more

January 17, 2022January 17, 2022

1st RRMS Patient Enrolled in ENSURE-2 Trial of Oral IMU-838

Multiple Sclerosis News Today

The ENSURE-2 Phase 3 clinical trial, evaluating Immunic Therapeutics’ experimental oral therapy IMU-838 (vidofludimus calcium) in adults with relapsing-remitting multiple sclerosis (RRMS), has enrolled its first patient. The milestone, announced ... Read more

January 14, 2022January 15, 2022

Epstein-Barr Virus May be Leading Cause of MS, Raising Risk by 32 Times

Multiple Sclerosis News Today

Infection with the common Epstein-Barr virus (EBV) increases the risk of developing multiple sclerosis (MS) by 32 times — the strongest link yet — according to a study looking at two ... Read more

January 14, 2022January 14, 2022

aTyr’s Immunomodulatory Therapy ATYR1923 Wins Orphan Drug Status

Sarcoidosis News

The U.S. Food and Drug Administration has granted orphan drug designation to aTyr Pharma’s experimental immunomodulatory molecule ATYR1923 for the treatment of sarcoidosis. The therapy is being developed for severe inflammatory diseases ... Read more

January 14, 2022January 14, 2022

AcuraStem’s AS-202 for ALS Shows Promise in Preclinical Studies

ALS News Today

AcuraStem’s experimental therapy AS-202 significantly reduced neurodegeneration and improved motor function and survival in a mouse model of aggressive amyotrophic lateral sclerosis (ALS) associated with toxic clumps of the TDP-43 ... Read more

January 14, 2022January 14, 2022

CTEPH Study Ties Lung Arterial Obstruction to Airflow Obstruction

Pulmonary Hypertension News

Increased lung arterial obstruction is significantly associated with greater airflow obstruction in people with chronic thromboembolic pulmonary hypertension (CTEPH) and without a smoking history, a study shows. Notably, airflow obstruction, ... Read more

January 13, 2022January 13, 2022

CLN6 Mutations May Lead to Batten Without Vision Loss, 3 Cases Show

Batten Disease News

Mutations in the CLN6 gene — which have been associated with different forms of Batten disease — may lead to the development of an atypical form of juvenile Batten that ... Read more

January 13, 2022January 13, 2022

Phase 1/2 Trial Set to Test RGX-202, Gene Therapy for Duchenne MD

Muscular Dystrophy News

The U.S. Food and Drug Administration (FDA) has approved Regenxbio’s request to launch a Phase 1/2 clinical trial in the U.S. evaluating the safety and efficacy of RGX-202, its experimental ... Read more

January 12, 2022January 12, 2022

Centogene Launches CentoCloud Tool for Rare Disease Diagnostics

AADC News

Centogene has launched CentoCloud, a cloud-based platform designed to allow decentralized analysis and interpretation of an individual’s genetic data, thereby providing fast diagnostic insights and medical reports. Reported to be ... Read more

January 12, 2022January 12, 2022

Benefits of Roctavian, Hem A Gene Therapy, Holding for 2 or More Years

Hemophilia News Today

A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) continues to prevent bleeds and the need for preventive treatment, or prophylaxis, over at least two years in men ... Read more

January 12, 2022January 13, 2022

Same C5a Complement Protein Mechanisms Found in aHUS, COVID-19

aHUS News, COVID-19 News

C5a, a terminal product of the complement system, can, by itself, promote blood vessel dysfunction and the subsequent formation of blood clots seen in people with atypical hemolytic uremic syndrome (aHUS), ... Read more

January 10, 2022January 10, 2022

FDA Expands MRI Compatibility With Spinal Stimulation for Pain

Multiple Sclerosis News Today

The U.S. Food and Drug Administration (FDA) has approved Abbott’s request to expand the magnetic resonance imaging (MRI) compatibility with its Proclaim XR spinal cord stimulation system with Octrode leads, ... Read more

January 10, 2022January 10, 2022

US Medical Data Show Higher Mortality in SMA Patients Before DMT Era

SMA News Today

Before the emergence of Spinraza (nusinersen) — the first disease-modifying therapy (DMT) approved for spinal muscular atrophy (SMA) — patients of all ages and types of the disease showed greater ... Read more

January 10, 2022January 10, 2022

Pridopidine Arm of HEALEY ALS Platform Trial Fully Enrolled

ALS News Today

Prilenia Therapeutics announced the completion of patient enrollment in the pridopidine arm of the HEALEY ALS platform trial, the first study to simultaneously test multiple experimental therapies for amyotrophic lateral ... Read more

January 10, 2022January 10, 2022

Immune Dysfunction Common in Hypermobile EDS, Study Finds

Ehlers-Danlos News

Immune dysfunction — including abnormal activation of mast cells and antibody deficiency — is common among people with hypermobile Ehlers–Danlos syndrome (hEDS) and those with hypermobile spectrum disorder (HSD), according ... Read more