The ALS Association announced the largest single gift in its history from the estate of the late philanthropist Hugh Hoffman. The $58 million gift is specifically designated to advancing promising ALS therapies, developing new or strengthening existing ALS clinics, and enhancing access to ALS care for people living with ALS.

The researchers detected abnormal proteins in the spinal fluid of people with ALS and frontotemporal dementia (FTD) that were linked to a lack of functioning TDP-43. These abnormal proteins could be used to help improve diagnosis and guide the development of novel therapies.

An experimental drug known as SAR443820 or DNL788 being developed by Denali Therapeutics and its partner Sanofi did not affect participants’ ALSFRS-R scores during the phase 2 HIMALAYA study, according to documents filed with the Securities and Exchange Commission. Detailed study results were not provided, but the filing noted Sanofi plans to present safety and efficacy data at an upcoming scientific conference.

The recommendation is for “marketing authorisation under exceptional circumstances. This allows patients access a drug, but indicates that the applicant was unable to provide comprehensive data either because there are very few people with the disease, the collection of complete efficacy and safety information would be unethical, or there are gaps in the scientific knowledge.

More News:

Clene Analyzes Survival Data Gathered from Two CNM-Au8 Expanded Access Programs

NeuroSense Releases Additional Positive Results from Small Phase 2b Trial of PrimeC

Transposon Therapeutics’ TPN-101 Has Effect on Key Biomarkers Based on Interim Phase 2 C9orf72-Related ALS/FTD Study Results

VA Opens First Cell Therapy Trial for Veterans with ALS

Experimental Gene Therapy CTx1000 Prevents Buildup of ALS/FTD Protein
TDP-43 in Mice

ALS Focus is a survey program developed by and for people living with ALS and their caregivers. Through ALS Focus research, the ALS Association scientifically measures the preferences, needs and experiences of people living with ALS and ALS caregivers to promote change in ALS research, policy, and care. All participants must be at least 18 years old, live in the United States and be able to read and understand English.

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Enhancing Mobility, Communication, and Quality of Life through Assistive Technology Research

Barnett Drug Development Program is Helping Six New Therapies Move Closer to Clinical Trials

The Generous Son of an ALS Parent Elevates the Fight Against ALS

Researcher Spotlight: Annie Collins, 2023 Milton Safenowitz Postdoctoral Fellow

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