Neurological disorders

Zynerba Pharmaceuticals Announces Presentation of Efficacy and Safety Findings in Children and Adolescents with Autism Spectrum Disorder (ASD) and Related Disorders at the 2021 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting

Retrieved on: 
Thursday, June 3, 2021

ET in poster session II at the 2021 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting.

Key Points: 
  • ET in poster session II at the 2021 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting.
  • A copy of the poster is available on the Zynerba corporate website at http://zynerba.com/publications/ .
  • The poster titled, ZYN002 Cannabidiol Transdermal Gel: Efficacy and Safety Findings in Children and Adolescents With Autism Spectrum Disorder (ASD) and Related Disorders, shows that Zygel may provide important clinical promise across a spectrum of endpoints, including, behavior, seizure reduction and sleep.
  • The data are from two open-label Phase 2 trials and one double-blind placebo-controlled trial when added to standard of care in children and adolescents.

Cognoa Receives FDA Marketing Authorization for First-of-its-kind Autism Diagnosis Aid

Retrieved on: 
Wednesday, June 2, 2021

The AI-based device is the first FDA-authorized diagnosis aid designed to help physicians to diagnose autism in the primary care setting.

Key Points: 
  • The AI-based device is the first FDA-authorized diagnosis aid designed to help physicians to diagnose autism in the primary care setting.
  • While autism can be reliably diagnosed in children as early as 18 months, the average age of autism diagnosis has remained at 4 to 5 years old for decades.
  • Disparities in autism diagnosis are largely due to the lack of female and diverse representation in autism research to date.
  • The FDA marketing authorization has been granted following a successful pivotal study in which the device exceeded study endpoints.

NeuBase Therapeutics Announces Speakers for Virtual R&D Day Taking Place on June 8th

Retrieved on: 
Wednesday, June 2, 2021

NeuBase will present new data for the Companys pipeline including myotonic dystrophy type 1 (DM1), Huntingtons disease (HD), and its new oncology program.

Key Points: 
  • NeuBase will present new data for the Companys pipeline including myotonic dystrophy type 1 (DM1), Huntingtons disease (HD), and its new oncology program.
  • Using the Companys proprietary PATrOL platform, NeuBase is designing precision genetic medicines to activate, silence, or edit genes, as appropriate, depending on the underlying genetic mutation driving disease.
  • This unifying platform capability is uniquely positioned to create a scalable pipeline of new medicines to address previously untreatable diseases.
  • Dr. Friedlander is an elected member of the prestigious American Society for Clinical Investigation and the Association of American Physicians.

Venture Construction Group of Florida Supports ALS® Awareness Month With Statewide Premier Sponsorship

Retrieved on: 
Wednesday, June 2, 2021

BOCA RATON, Fla., June 2, 2021 /PRNewswire-PRWeb/ -- A major advocate for ALS research and fighting for a cure, Venture Construction Group of Florida (VCGFL) is proud to be the Premier 2021 Statewide Partner of the ALS Association Florida Chapter to raise funds and awareness.

Key Points: 
  • BOCA RATON, Fla., June 2, 2021 /PRNewswire-PRWeb/ -- A major advocate for ALS research and fighting for a cure, Venture Construction Group of Florida (VCGFL) is proud to be the Premier 2021 Statewide Partner of the ALS Association Florida Chapter to raise funds and awareness.
  • We're dedicated to supporting ALS research and finding a cure," said Stephen Shanton, CEO and president of Venture Construction Group of Florida (VCGFL).
  • "The programs and services The ALS Association Florida Chapter provides wouldn't be possible without philanthropic leaders like Venture Construction Group of Florida.
  • VCGFL is an award-winning construction and restoration company committed to giving back and supports numerous ALS organizations and programs each year.

Lighthouse Autism Center Announces New Center in Valparaiso, IN

Retrieved on: 
Tuesday, June 1, 2021

SOUTH BEND, Ind., June 1, 2021 /PRNewswire-PRWeb/ --Lighthouse Autism Center, an ABA therapy center for children with autism, recently announced its expansion into northwest Indiana coming this summer.

Key Points: 
  • SOUTH BEND, Ind., June 1, 2021 /PRNewswire-PRWeb/ --Lighthouse Autism Center, an ABA therapy center for children with autism, recently announced its expansion into northwest Indiana coming this summer.
  • Beginning in July, Lighthouse Autism Center will offer autism therapy services at its newest center in Valparaiso, Indiana.
  • Gregg and Sandy Maggioli, founders of Lighthouse Autism Center and parents to a child with autism are excited to expand services into northwest Indiana.
  • Lighthouse Autism Center is an ABA therapy center operated by parents of a child with autism serving Indiana and Michigan.

Alnylam Completes Enrollment in APOLLO-B Phase 3 Study of Patisiran in Transthyretin-Mediated (ATTR) Amyloidosis Patients with Cardiomyopathy

Retrieved on: 
Tuesday, June 1, 2021

Enrollment was completed with over 300 ATTR amyloidosis patients across 90 sites in more than 20 countries.

Key Points: 
  • Enrollment was completed with over 300 ATTR amyloidosis patients across 90 sites in more than 20 countries.
  • The APOLLO-B trial was initiated based on encouraging data from previous studies that support further investigation of patisiran in ATTR amyloidosis patients with cardiomyopathy.
  • Specifically, encouraging results were obtained from the landmark Phase 3 APOLLO study in hATTR amyloidosis patients with polyneuropathy on a number of exploratory cardiac endpoints.
  • The APOLLO-B study is a Phase 3, randomized, double-blind, placebo-controlled multicenter global study designed to evaluate the efficacy and safety of patisiran in patients with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, which enrolled over 300 adult patients with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy.

National ALS Registry Honors Lou Gehrig

Retrieved on: 
Friday, May 28, 2021

ATLANTA, May 28, 2021 /PRNewswire/ -- Join the National ALS Registry in honoring baseball legend Lou Gehrig and raising awareness of the disease that bears his name.

Key Points: 
  • ATLANTA, May 28, 2021 /PRNewswire/ -- Join the National ALS Registry in honoring baseball legend Lou Gehrig and raising awareness of the disease that bears his name.
  • On June 2nd Major League Baseball (MLB) will celebrate the inaugural "Lou Gehrig Day" as a day to commemorate the life and legacy of Lou Gehrig.
  • The National ALS Registry, which is part of the Centers for Disease Control and Prevention (CDC) and the Agency for Toxic Substances and Disease Registry (ATSDR), honors Lou Gehrig and aims to help the search for a cure for the disease by collecting, managing, and analyzing data about people with ALS.
  • To learn more about ALS and the work of the National ALS Registry, please visit cdc.gov/als
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Jacob Family Pledges $250,000 to the Foundation for Angelman Syndrome Therapeutics

Retrieved on: 
Thursday, May 27, 2021

AUSTIN, Texas, May 27, 2021 /PRNewswire/ --Austin-based philanthropists, Ryan and Annie Jacob, announce a $250,000 commitment to the Foundation for Angelman Syndrome Therapeutics (FAST) through a new donation matching program.

Key Points: 
  • AUSTIN, Texas, May 27, 2021 /PRNewswire/ --Austin-based philanthropists, Ryan and Annie Jacob, announce a $250,000 commitment to the Foundation for Angelman Syndrome Therapeutics (FAST) through a new donation matching program.
  • Founded in 2008, FAST is dedicated to finding a cure for Angelman syndrome by funding a robust and aggressive research agenda.
  • FAST's research initiatives are focused on identifying potentially transformative therapeutics to address the symptoms of Angelman syndrome.
  • FAST is committed to assisting individuals living with Angelman syndrome to realize their full potential and quality of life through effective therapeutics.

ProMIS Neurosciences appoints renowned scientist, Dr. David Wishart as Chief Physics Officer

Retrieved on: 
Thursday, May 27, 2021

ProMIS has leveraged its industry leading technology platform to create a portfolio of antibody, intrabody and vaccine candidates that are highly selective for the misfolded protein aggregates driving pathogenesis, stated Dr. Neil Cashman, ProMIS Chief Scientific Officer (CSO).

Key Points: 
  • ProMIS has leveraged its industry leading technology platform to create a portfolio of antibody, intrabody and vaccine candidates that are highly selective for the misfolded protein aggregates driving pathogenesis, stated Dr. Neil Cashman, ProMIS Chief Scientific Officer (CSO).
  • As Chief Physics Officer, Dr. Wishart will play a pivotal role to further develop and expand the application of our proprietary platform to the biology of additional misfolded protein diseases.
  • I would also like to take this opportunity to sincerely thank Dr. Steven Plotkin for his outstanding prior service to the company as the ProMIS Chief Physics Officer since its inception.
  • ProMISs unique capability is our unrivalled antibody discovery platform, and Dr. Wishart has already added immeasurably to that capability, said Executive Chairman Eugene Williams.

Alector to Host Virtual Key Opinion Leader Event on Frontotemporal Dementia on June 11, 2021

Retrieved on: 
Wednesday, May 26, 2021

SOUTH SAN FRANCISCO, Calif., May 26, 2021 (GLOBE NEWSWIRE) -- Alector, Inc.(Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, today announced that it will host a virtual key opinion leader event focused on frontotemporal dementia due to a progranulin gene mutation (FTD-GRN) onFriday, June 11, 2021 at10:30 a.m.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., May 26, 2021 (GLOBE NEWSWIRE) -- Alector, Inc.(Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, today announced that it will host a virtual key opinion leader event focused on frontotemporal dementia due to a progranulin gene mutation (FTD-GRN) onFriday, June 11, 2021 at10:30 a.m.
  • Alector management will also provide an overview of the ongoing development program for AL001 in FTD-GRN.
  • A replay will be available on the Alector website for 30 days following the event.
  • AL001 has received Orphan Drug designation for the treatment of FTD and Fast Track designation for the treatment of FTD-GRN from theU.S.