A branch of the European Medicines Agency (EMA) has recommended that Prilenia’s investigational therapy pridopidine be designated an orphan medicine to treat people with amyotrophic lateral sclerosis (ALS). Medicines with the potential to become safe and effective treatments for rare, life-threatening, or chronically debilitating conditions affecting no more than one…
“It wasn’t me she was foolin’, ‘Cause she knew what was she was doin’, When she told me how to walk this way.” Run-DMC’s hip hop adaptation of the Aerosmith classic “Walk This Way” conjures up images of every therapy session I’ve had during the ALS portion of my…
At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…
The other day while watching TV, I heard the newscaster announce, “We’re in the ‘new normal’ folks, and everybody wants to get out and travel again!” “Well, I don’t,” I thought, and just as quickly wondered, “Am I the only one who doesn’t feel comfortable yet to go out…
Note: This story was updated June 30, 2021, to clarify that Exservan is placed on top of the tongue, not under it. Specialty pharmacy services for Exservan (riluzole), a twice-daily film that dissolves when placed on the tongue and approved to treat amyotrophic lateral sclerosis…
“He wants to give the answers but his words are only Answers to nothing … They think this is the answer, But their thoughts are only Answers to nothing.” — Midge Ure “And the vision that was planted in my brain Still remains Within the sound of silence.” —…
Largely due to going virtual this year, the annual HayesDavidson 5K (HD5K) charity run met its goal of raising £40,000 (nearly $56,000) for the Motor Neurone Disease (MND) Association. The event previously was held in Hyde Park in London. However, the switch to the digital format enabled more…
Mutations in the FUS gene, a common cause of amyotrophic lateral sclerosis (ALS), impair the regeneration and growth of motor neuron extensions toward muscle cells, according to a study using a new miniaturized human model of neuron-muscle interactions. These deficits, along with the reduced number of nerve-motor connections, were lessened by blocking…
Most unpaid caregivers in the U.K. have not had any breaks from their caregiving role during the COVID-19 pandemic, with nearly three-quarters reporting feeling exhausted, stressed, and anxious, according to a survey of more than 2,800 caregivers conducted by Carers UK. During the pandemic, carers lost a mean…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
