News

November 24, 2020November 24, 2020

Dosing Begins in Phase 2 Study of Pegcetacoplan in ALS Patients

News

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 ... Read more

November 23, 2020November 23, 2020

50K-signature Petition Calling for Swift Approval of AMX0035 Given FDA, Amylyx

News

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) ... Read more

November 20, 2020November 20, 2020

FDA Grants Orphan Drug Designation to SLS-005 for Slowing ALS Progression

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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 ... Read more

November 18, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

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 ... Read more

November 18, 2020November 24, 2020

NurOwn Fails to Significantly Slow Disease Progression in ALS Patients, Top-line Data Show

News

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. ... Read more

November 17, 2020November 17, 2020

Radicava’s Effectiveness for ALS Not Established, Real-life Use Suggests

News

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 ... Read more

November 16, 2020November 16, 2020

UK Charities Offer Counseling to Children Confronting ALS, MND in Family

News

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 ... Read more

November 13, 2020November 13, 2020

Wearable Facial Sensor May Help ALS Patients More Easily Communicate

News

A lightweight, wearable sensor is being developed that can detect minute facial movements and translate them into messages, potentially helping people with amyotrophic lateral sclerosis (ALS) communicate more easily. ... Read more

November 12, 2020November 12, 2020

Combo Bone Marrow Cell Transplant Seen to Benefit Mice in ALS Model

News

Transplanting a combination of two types of modified bone marrow cells eased motor deficits and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS), researchers reported. Their study, ... Read more

November 11, 2020November 11, 2020

Global Phase 3 Study of Oral Edaravone to Treat ALS Fully Enrolled

News

The Phase 3 clinical trial investigating an oral suspension formulation of edaravone (MT-1186) in treating amyotrophic lateral sclerosis (ALS) is now fully enrolled, the therapy’s developer, Mitsubishi Tanabe Pharma America, announced. ... Read more

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