Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
A gene therapy that might treat familial amyotrophic lateral sclerosis (ALS), an inherited form of the disease, was recently granted orphan drug status by the European Medicines Agency (EMA), an award that carries incentives to promote potential treatments for rare diseases. The investigative therapy is in preclinical development…
Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough new law that aims to protect the EU’s 512 million citizens, including rare disease patients, from having their medical records misused, sold, or subject to extortion by hackers, third…
Clinigen Group recently partnered with Mitsubishi Tanabe Pharma Corporation to launch an early access program in Europe for Radicava (edaravone), an intravenous treatment for amyotrophic lateral sclerosis (ALS). The therapy is not yet approved in Europe, but the early access program will help make it available…
Voyager Therapeutics presented new data from a study in an animal model of its clinical candidate VY-SOD101, showing the therapy is effective as it targets the most common cause of familial amyotrophic lateral sclerosis (ALS). The presentation, along with another preclinical program data presentation on a Huntington’s disease therapy…
There’s no doubt about it — living with ALS has sure slowed me down! And if you have ALS, you probably feel your life slowing down as well. It’s good to know that you’re not alone. Moving, eating, and talking more slowly, and feeling fatigued are common…
A new mouse model developed by researchers at Karolinska Institute in Sweden revealed a molecular mechanism that may contribute to the development of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) or multiple sclerosis. The mouse model was described in the study, “Fatal demyelinating disease…
The protein TRIF can prevent resident immune cells of the brain from activating abnormally and slow the progression of amyotrophic lateral sclerosis (ALS), researchers in Japan working in a mouse model of the disease report. Their work, “Innate immune adaptor TRIF deficiency accelerates disease progression of ALS mice with…
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
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