Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

My mind can easily spiral into anxiety if I let it. I got selected for jury duty. Along with my request to be excused because I’m my husband’s caregiver, I included doctors’ notes stating that my husband, Todd, is paralyzed. Apparently, that wasn’t enough, because I received a second round…

“Pleased to meet you, hope you guess my name.” — Keith Richards and Mick Jagger, “Sympathy for the Devil” Last week, I experienced an abysmal customer service episode. Lasting the better part of a full workday, the bitter crescendo left…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Mitsubishi Tanabe Pharma America announced changes to enhance its out-of-pocket assistance program for people with amyotrophic lateral sclerosis (ALS) using Radicava (edaravone). The changes, aimed at making the program easier and more accessible, include a new “hybrid” card meant to help patients obtain appropriate pharmacy and medical co-pay…

Mexiletine, a heart medication used off-label to treat muscle cramps due to amyotrophic lateral sclerosis (ALS), was found to be well-tolerated and to stabilize the “hyperexcitability” of motor neurons in a small group of ALS patients in a pilot Phase 2 trial. Data from the study (NCT02781454)…

The European Medicines Agency has granted orphan drug designation to PrimeC, an investigational combination therapy for amyotrophic lateral sclerosis (ALS). PrimeC, developed by NeuroSense Therapeutics, also has received orphan drug designation in the United States. The designation, both in the U.S. and Europe, encourages drugmakers to develop…

Mitsubishi Tanabe Pharma America (MTPA) is taking proactive measures to address patient safety in its REFINE-ALS biomarker study during the COVID-19 pandemic. The company announced revisions to the study’s protocol that give participants the option to participate virtually, through the use of telemedicine (medical care delivered virtually) and…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Nearly half of all patients in a Phase 2 trial of CNM-Au8, Clene Nanomedicine’s lead candidate for the treatment of amyotrophic lateral sclerosis (ALS), showed improvements in the health and functioning of their motor neurons, according to the interim trial data. These data are still blinded,…