About Anne D Pham

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So far Anne D Pham has created 68 blog entries.

ALS Finding a Cure Call for Proposals in Early Diagnostics

2022-10-20T19:48:18+00:00

ALS Finding a Cure Call for Proposals in Early Diagnostics Shockingly, more than 150 years after the disease was first defined, there are currently no universally accepted diagnostic approaches for ALS. In order to receive an ALS diagnosis patients must first undergo a battery of tests designed to rule out other diagnoses. This is highly problematic because it can take a year for an ALS patient to receive the correct diagnosis (and sometimes even longer). While these individuals are searching for a name for their affliction, they are frequently bounced to multiple doctors, can receive unnecessary (and expensive) surgical procedures [...]

ALS Finding a Cure Call for Proposals in Early Diagnostics2022-10-20T19:48:18+00:00

ALS Finding a Cure Providing the Initial Funding for Amylyx

2022-10-03T00:59:42+00:00

ALS Finding a Cure Providing the Initial Funding for Amylyx In every journey of discovery, certain days distinguish themselves as fundamental to achieving the ultimate objective. Yesterday was one of those days in the fight against ALS, Lou Gehrig’s Disease, with the FDA’s approval of Amylyx’s new drug, Relyvrio, the first new ALS drug in over five years. ALS (amyotrophic lateral sclerosis) is a devastating disease of the nervous system which robs patients of their muscle control and eventually their ability to breathe on their own.  While rare, there is no known cure, and it famously struck down Lou [...]

ALS Finding a Cure Providing the Initial Funding for Amylyx2022-10-03T00:59:42+00:00

Publications related to Answer ALS, a project ALS FAC helped fund

2022-09-28T00:26:41+00:00

Publications related to Answer ALS, a project ALS FAC helped fund 1. Baxi, E. G. et al. Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines. Nat Neurosci 1-12 (2022) doi:10.1038/s41593-021-01006-0. 2. Zhang, S. et al. Genome-wide identification of the genetic basis of amyotrophic lateral sclerosis. Neuron (2022) doi:10.1016/j.neuron.2021.12.019. 3. Gilley, J. et al. Enrichment of SARM1 alleles encoding variants with constitutively hyperactive NADase in patients with ALS and other motor nerve disorders. Medrxiv 2021.06.17.21258268 (2021) doi:10.1101/2021.06.17.21258268. 4. Ramamoorthy, D. et al. Identifying Patterns of ALS Progression from Sparse Longitudinal Data. Medrxiv 2021.05.13.21254848 (2021) doi:10.1101/2021.05.13.21254848. 5. Coyne, A. N. et al. Nuclear accumulation of CHMP7 [...]

Publications related to Answer ALS, a project ALS FAC helped fund2022-09-28T00:26:41+00:00

Independent FDA Advisory Panel Recommends Approving ALS Drug

2022-09-13T18:04:22+00:00

A panel of independent advisers to the US Food and Drug Administration voted Wednesday (September 7) to recommend the agency approve a novel therapy to treat amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease. The drug, called AMX0035, had previously been rejected by the same group earlier in the year, but new evidence presented by the company that developed it, Massachusetts-based Amylyx Pharmaceuticals, prompted a surprising reversal that also comes amid an aggressive lobbying campaign by ALS patients and their families. Read More...

Independent FDA Advisory Panel Recommends Approving ALS Drug2022-09-13T18:04:22+00:00

Healey & AMG Center, ALS Finding a Cure® and FightMND announce the 2022 ALS Scholars in Therapeutics Recipients

2022-08-25T12:23:38+00:00

Class of 2022 Boston—The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH), ALS Finding a Cure® and FightMND selected three recipients for their global two-year ALS Scholars in Therapeutics program. The program received an outstanding pool of global applications from 4 countries. This year’s selected recipients listed alphabetically are Dylan Galloway, PhD of the Washington University School of Medicine, Jacob Mann, PhD of Northwestern University Feinberg School of Medicine, and Roberta Piovesana, PhD of the University of Montreal. Dylan Galloway, PhD Dylan Galloway, PhD is recognized for his research in identifying microRNAs as novel regulators of [...]

Healey & AMG Center, ALS Finding a Cure® and FightMND announce the 2022 ALS Scholars in Therapeutics Recipients2022-08-25T12:23:38+00:00

AI Finds Potential Drug Targets for Amyotrophic Lateral Sclerosis

2022-07-07T19:45:26+00:00

Scientists have identified several potential therapeutic targets for amyotrophic lateral sclerosis (ALS) using an artificial intelligence (AI)-powered target discovery engine to analyze dysregulated gene expression in large, publicly available ALS datasets.

AI Finds Potential Drug Targets for Amyotrophic Lateral Sclerosis2022-07-07T19:45:26+00:00

ALS Finding a Cure® funded Amylyx’s preclinical and early clinical work

2022-06-15T18:43:49+00:00

Health Canada decision signifies the first global regulatory approval for Amylyx and the first new therapy for ALS approved in Canada since 2018 ALBRIOZA (also known as AMX0035) is an oral fixed-dose combination therapy that may reduce neuronal cell death as a stand-alone therapy or when added to existing treatments In a clinical trial, ALBRIOZA significantly slowed disease progression and loss of functional decline in people living with ALS Detailed data from the CENTAUR clinical trial were published in the New England Journal of Medicine,Muscle & Nerve, and the Journal of Neurology, Neurosurgery and Psychiatry

ALS Finding a Cure® funded Amylyx’s preclinical and early clinical work2022-06-15T18:43:49+00:00

ALS Finding a Cure® funded the SOD1 1/2 life work used in the Tofersen study

2022-06-15T18:44:53+00:00

Twelve-month data show that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength and quality of life Tofersen also led to robust and sustained reductions in neurofilament, a marker of neurodegeneration SOD1-ALS is a rare, progressive and fatal genetic form of the disease leading to loss of everyday functions and affecting approximately 2% of people with ALS

ALS Finding a Cure® funded the SOD1 1/2 life work used in the Tofersen study2022-06-15T18:44:53+00:00

ALS Finding a Cure® funded Eledon’s early clinical work for Tegoprubart when the drug was still with ALS TDI

2022-06-15T18:44:42+00:00

IRVINE, Calif., May 31, 2022 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (Nasdaq: ELDN), a patient-focused clinical stage biopharmaceutical company committed to the development of innovative and impactful treatments for organ and cell transplantation, autoimmune conditions, and neurodegenerative disease, today announced topline results from a Phase 2a clinical trial of tegoprubart (formerly AT-1501) in patients with amyotrophic lateral sclerosis (ALS).1

ALS Finding a Cure® funded Eledon’s early clinical work for Tegoprubart when the drug was still with ALS TDI2022-06-15T18:44:42+00:00

ALS Association Supports Study of SARM1 Variants

2022-04-19T13:26:30+00:00

The ALS Association, in collaboration with ALS Finding a Cure, recently awarded $400,000 to support research into the role SARM1 gene mutations play in the development and progression of ALS, with the ultimate goal of using an improved understanding of SARM1 to develop new treatments. The research will be led by Dr. Jemeen Sreedharan at King’s College London and Dr. Michael Coleman at Cambridge University.

ALS Association Supports Study of SARM1 Variants2022-04-19T13:26:30+00:00
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