Anne D Pham – Page 5 – ALS Finding a Cure®

ALSFAC-Funded Project Announces Successful ALS Clinical Trial and High-Profile Publication
Gallery

ALSFAC-Funded Project Announces Successful ALS Clinical Trial and High-Profile Publication

Anne D Pham2021-09-07T16:20:37+00:00

ALSFAC-Funded Project Announces Successful ALS Clinical Trial and High-Profile Publication Amylyx, a start-up biotech company developing new treatments for ALS supported by ALS Finding a Cure®, and Mass General Hospital, announced the publication of results from its successful pivotal stage ALS clinical trial in the prestigious New England Journal of Medicine (NEJM). ALS Finding A Cure® provided two grants to Amylyx: the first in collaboration with Cure Alzheimer’s Disease to support required preclinical toxicology studies, and the second to fund this trial in collaboration with The ALS Association as part of the ALS ACT initiative. The clinical trial, known as [...]

ALSFAC-Funded Project Announces Successful ALS Clinical Trial and High-Profile PublicationAnne D Pham2021-09-07T16:20:37+00:00

ALSFAC Funded Research Turns Off Key Gene in Families with ALS
Gallery

ALSFAC Funded Research Turns Off Key Gene in Families with ALS

Anne D Pham2021-09-07T16:20:37+00:00

ALSFAC Funded Research Turns Off Key Gene in Families with ALS Researchers at the University of Massachusetts Medical School and Massachusetts General Hospital report progress on a potential new weapon against the fatal nerve disease ALS. Read More Report Progress

ALSFAC Funded Research Turns Off Key Gene in Families with ALSAnne D Pham2021-09-07T16:20:37+00:00

Team of Five Labs Launches Novel Attack on ALS

Anne D Pham2021-09-07T16:20:37+00:00

Team of Five Labs Launches Novel Attack on ALS Amyotrophic Lateral Sclerosis (ALS) can arise from aberrant genes. A group of five Brown University professors proposes that a cure may also come from aberrant genes – genetic mutations that suppress ALS. A new research grant supports their comprehensive investigation of ALS in flies, worms, mice and human cells.

Team of Five Labs Launches Novel Attack on ALSAnne D Pham2021-09-07T16:20:37+00:00

Progress Coming in Amyotrophic Lateral Sclerosis, but Rigorous Studies Needed

Anne D Pham2021-09-07T16:20:37+00:00

Progress Coming in Amyotrophic Lateral Sclerosis, but Rigorous Studies Needed The community of individuals living with amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, has endured many false hopes for promising therapies and is still waiting for the first disease-modifying drug. Nevertheless, "we have made huge progress in the last decade in ALS," Merit Cudkowicz, professor of neurology at Harvard Medical School and chief of the neurology service and director of the MDA ALS clinic at Massachusetts General Hospital, told participants in an integrated neuroscience session on ALS at the American Academy of Neurology... READ MORE [...]

Progress Coming in Amyotrophic Lateral Sclerosis, but Rigorous Studies NeededAnne D Pham2021-09-07T16:20:37+00:00

Johns Hopkins University, Cedars-Sinai and Massachusetts General Hospital Announce Groundbreaking ALS Research Project of Answer ALS

Anne D Pham2022-03-09T16:33:09+00:00

Johns Hopkins University, Cedars-Sinai and Massachusetts General Hospital Announce Groundbreaking ALS Research Project of Answer ALS Research Designed to Fully Understand and Defeat ALS; “Big Data” Technology Will Drive Unprecedented Analysis Capability $20 Million in Funding from Committed Team of Donors, including the National Football League, the PGA TOUR, ALS Finding a Cure (a project of the Leandro P. Rizzuto Foundation), Travelers, The Fishman Family and The Bari Lipp Foundation BALTIMORE, LOS ANGELES and BOSTON, September 9, 2015 – Johns Hopkins University’s Robert Packard Center for ALS Research, Cedars-Sinai Medical Center’s Regenerative Medicine Institute and the Massachusetts General Hospital Neurological [...]

Johns Hopkins University, Cedars-Sinai and Massachusetts General Hospital Announce Groundbreaking ALS Research Project of Answer ALSAnne D Pham2022-03-09T16:33:09+00:00

Karyopharm Advancing Neuroprotective SINE™ Compounds in Amyotrophic Lateral Sclerosis (ALS)

Anne D Pham2021-09-07T16:20:38+00:00

Karyopharm Advancing Neuroprotective SINE™ Compounds in Amyotrophic Lateral Sclerosis (ALS) Preclinical efficacy of SINE™ compounds validated in journal Nature publication– Presentations of ALS abstracts at upcoming AAN and SfN meetings – Collaborator grant funding supports Karyopharm’s ALS research– Newton, Mass. – September 3, 2015 – Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, today reported that its SINE™ nuclear transport compounds are being evaluated for the treatment of Amyotrophic Lateral Sclerosis (ALS) and that preclinical data abstracts will be presented at two upcoming neuroscience meetings. A recent publication appearing in the journal Nature confirmed that nuclear transport is disrupted in [...]

Karyopharm Advancing Neuroprotective SINE™ Compounds in Amyotrophic Lateral Sclerosis (ALS)Anne D Pham2021-09-07T16:20:38+00:00

Treg Study: Therapeutic Goal – To Increase Neuroprotective FoxP3 Tregs in ALS Patients

Anne D Pham2021-06-17T15:13:54+00:00

Treg Study: Therapeutic Goal – To Increase Neuroprotective FoxP3 Tregs in ALS Patients Research Summary Principle Investigator: Stanley Appel MD This study may lead to Treg cell therapy for symptom management or potential slowing of disease progression in ALS and related disorders. This study is to isolate T regulatory (Treg) cells from the blood using leukapheresis, and expand T regulatory (Treg) cells in the laboratory in up to twelve patients with ALS. Leukapheresis is a laboratory procedure in which white blood cells are separated from a sample of blood. Once separated, the remainder of the blood is returned to the [...]

Treg Study: Therapeutic Goal – To Increase Neuroprotective FoxP3 Tregs in ALS PatientsAnne D Pham2021-06-17T15:13:54+00:00

Development of AAV-Mediated SOD1 Gene Silencing Therapy in ALS

Anne D Pham2021-06-17T15:12:58+00:00

Development of AAV-Mediated SOD1 Gene Silencing Therapy in ALS Research Summary Principle Investigators: Robert Brown D.Phil., MD & Merit Cudkowicz MD About 10% of ALS cases are familial. Most of the known genetic defects in familial ALS act by triggering one or more toxic processes that impair the viability of motor neurons, leading to motor neuron death. This project will use viruses to introduce reagents into the brain and spinal cord that block the mutant genes from triggering toxicity. This is accomplished by turning off the activity of those genes using new technologies for so-called “gene silencing.” The viruses are used [...]

Development of AAV-Mediated SOD1 Gene Silencing Therapy in ALSAnne D Pham2021-06-17T15:12:58+00:00

Silencing of ALS-Causing Mutant Genes

Anne D Pham2021-06-17T15:11:56+00:00

Silencing of ALS-Causing Mutant Genes Research Summary Principle Investigators: Robert Brown D.Phil., MD & Merit Cudkowicz MD About 10% of cases of ALS run in families and are caused by mutant genes, including the first-identified ALS gene known as superoxide dismutase 1 (SOD1). In most cases, the mutant genes make a mutant, toxic gene product, which in the case of SOD1 is a protein with a propensity to misfold. Data also argue that non-mutant forms of these proteins may also play a role in non-familial ALS. Several lines of study indicate that when SOD1 is implicated in ALS, the goal is [...]

Silencing of ALS-Causing Mutant GenesAnne D Pham2021-06-17T15:11:56+00:00

DIALS (Dominant Inherited ALS) Network Research Summary

Anne D Pham2021-06-17T15:10:53+00:00

DIALS (Dominant Inherited ALS) Network Research Summary Research Summary While evidence of a pre-symptomatic period has emerged for a number of neurodegenerative diseases, a similar precursor period prior to symptom development in ALS has yet to be defined, and pre-symptomatic biomarkers of ALS have yet to be discovered. As a result, researchers are unable to target people in the very earliest stages of ALS, when neurodegeneration is most nascent and may be most easily modified or halted. Current barriers to genetic testing in asymptomatic family members of people with ALS include insurance eligibility and cost. By using a funded research [...]

DIALS (Dominant Inherited ALS) Network Research SummaryAnne D Pham2021-06-17T15:10:53+00:00

About Anne D Pham