Investigational RNA Molecule May Slow ALS Progression
An investigational RNA molecule called an RNA aptamer halted the death of motor neurons and helped preserve motor function in…
Articles by Patricia Inácio, PhD
Spinogenix’s SPG302 Oral Therapy Named Orphan Drug by FDA
The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Spinogenix’s investigational oral molecule SPG302 for…
Advocates Can Try for Grants Ahead of Rare Disease Week on Capitol Hill
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
Phase 1/2 Trial of APB-102, Gene Therapy for Familial ALS, Cleared to Open
Clearance was given to open a Phase 1/2 trial evaluating APB-102, a potential gene therapy for amyotrophic lateral sclerosis (ALS)…
Global Phase 3 Trial of AMX0035 to Enroll 600 ALS Patients, Amylyx Says
A global Phase 3 trial of AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS) intends to…
Arimoclomol Fails to Show Efficacy in Phase 3 Trial, Topline Data Show
A Phase 3 clinical trial evaluating Orphazyme’s investigational oral therapy arimoclomol for amyotrophic lateral sclerosis (ALS) has failed to…
Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
Toyota, MND Association Team Up to Raise Funds, Awareness in UK
The Motor Neurone Disease (MND) Association and Toyota (GB), the U.K. division of Toyota, have established a partnership to raise…
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
Actimed Plans to Advance Treatment for Muscle Wasting in ALS
Actimed Therapeutics is developing S-oxprenolol to prevent muscle wasting and body mass loss due to amyotrophic lateral sclerosis (ALS), using…