A first patient has been dosed in the Phase 2 trial investigating pegcetacoplan (APL-2), Apellis Pharmaceuticals‘ candidate therapy for amyotrophic lateral sclerosis (ALS), the company and the Swedish Orphan Biovitrum (Sobi) announced. The potentially pivotal trial, called MERIDIAN (NCT04579666), is currently enrolling patients…
The ALS Association and I AM ALS have filed a petition with the U.S. Food and Drug Administration (FDA), calling on the regulatory agency and Amylyx Pharmaceuticals to make AMX0035 available to people with amyotrophic lateral sclerosis (ALS) as quickly as possible. Signed by more than 50,000 patients, their caregivers and family, the…
The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Seelos Therapeutics‘ SLS-005 (trehalose), a medication designed to slow the progression of amyotrophic lateral sclerosis (ALS), the company announced in a recent press release. The designation…
I cried as I listened to an episode of the “Everything Happens” podcast. Writer Samantha Irby described growing up with sick parents. When she was 9, her mom was diagnosed with multiple sclerosis, and her dad struggled with heart problems for years. They both died when she was…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
“Put me in, coach, I’m ready to play today, Look at me, I can be center field.” John Fogerty’s homage to an aspiring baseball player is reminiscent of my pre-ALS relationship with nearly any game of skill. It was the rare…
BrainStorm Cell Therapeutics’ cell-based therapy NurOwn failed to significantly slow disease progression in people with rapidly progressing amyotrophic lateral sclerosis (ALS), top-line data from a Phase 3 trial show. The therapy, however, did result in clinically meaningful responses in a pre-specified group of patients with early ALS…
As one of the moderators for the ALS News Today Forums, I get to welcome new members and learn a little bit about each person. Many of these new members are newly diagnosed patients. Besides wanting to learn as much as possible about ALS, they also tell me they…
A review of Radicava (edaravone), an approved amyotrophic lateral sclerosis (ALS) treatment, found the medication generally well tolerated in real-life use by patients in six countries, matching reports from clinical trials. But its effectiveness at delaying disease progression was less evident, with findings of greatest efficacy coming from Asia…
The Motor Neurone Disease (MND) Association and the children’s charity Barnardo’s have launched a counseling service for young people in the U.K. whose family is affected by amyotrophic lateral sclerosis (ALS) or another motor neurone disease (MND). The service is expected to open counseling help to these…
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