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Mashup Score: 0BrainStorm Withdraws BLA for ALS Therapy, Plans Phase IIIb Trial - 1 year(s) ago
After an FDA advisory committee voted 17-1 against approving NurOwn last month, BrainStorm executives Wednesday laid out a potential path forward for the experimental amyotrophic lateral sclerosis treatment.
Source: www.biospace.comCategories: Latest Headlines, Rare DiseaseTweet
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Mashup Score: 1Indo US Bridging RARE Summit 2023 - 1 year(s) ago
Boost global awareness at the IndoUS Bridging RARE Summit 2023!
Source: summit.indousrare.orgCategories: Latest Headlines, Rare DiseaseTweet-
Join @indousrare at the Bridging RARE Summit 2023, connecting the Indian diaspora for a discussion of #RareDisease research and #ClinicalTrials globally on October 29-30. Learn more here: https://t.co/cAKn0G0gmp #bridge4rare #bridgingrare
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Mashup Score: 0A Potential Gene Therapy Treatment for Bardet-Biedel Syndrome - 1 year(s) ago
Ciliopathies are a group of rare inherited genetic disorders and have long posed a significant challenge to the medical community.
Source: CheckRare.comCategories: Latest Headlines, Rare DiseaseTweet-
AXV101, the lead program developed by Axovia Therapeutics, is an adeno-associated virus (AAV9)-based gene therapy specifically targeting retinal dystrophy associated with bardet-biedel syndrome #BBS #checkrare https://t.co/968EOaxnQA
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Mashup Score: 0Educating Patient Advocates - 1 year(s) ago
Cheryl Petruk, co-founder of CACHEducation, talks about the need to properly educate patient advocates on advocacy fundamentals.
Source: CheckRare.comCategories: Latest Headlines, Rare DiseaseTweet-
Cheryl Petruk, co-founder of CACHEducation, talks about the need to properly educate patient advocates on the fundamentals of patient advocacy #checkrare #CACHEducation https://t.co/JeKKF9bdFD
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Mashup Score: 0Managing Cardiomyopathies in Lysosomal Disorders - 1 year(s) ago
Dr Ozlem Goker-Alpan and Dr John Jefferies discuss the role of the cardiologist and best practices for managing Lysosomal Disorders
Source: CheckRare.comCategories: Latest Headlines, Rare DiseaseTweet-
This CME/CE activity describes the pathophysiologies and management options for lysosomal disease patients with cardiomyopathies #checkrare #CME #cardiomyopathies https://t.co/oEdH8lGoT3 https://t.co/p1qkFNgve6
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Mashup Score: 0
Neurosense Therapeutics Ltd, a pioneering company in the field of neurodegenerative disease treatment, is revolutionizing the way we approach conditions
Source: beststocks.comCategories: Latest Headlines, Rare DiseaseTweet-
Neurosense Therapeutics https://t.co/iRF9evnT7v
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Mashup Score: 1New EAP makes CNM-Au8 available to ALS patients outside trials - 1 year(s) ago
The expanded access program should provide additional clinical data supporting the safety and effectiveness of CNM-Au8 toward its approval.
Source: alsnewstoday.comCategories: Latest Headlines, Rare DiseaseTweet-
The expanded access program should provide additional clinical data supporting the safety and effectiveness of CNM-Au8 toward its approval. https://t.co/z7uvUV0epf
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Mashup Score: 5Roadmap for C9ORF72 in Frontotemporal Dementia and Amyotrophic Lateral Sclerosis: Report on the C9ORF72 FTD/ALS Summit - 1 year(s) ago
Neurology and Therapy – A summit held March 2023 in Scottsdale, Arizona (USA) focused on the intronic hexanucleotide expansion in the C9ORF72 gene and its relevance in frontotemporal dementia (FTD)…
Source: link.springer.comCategories: Latest Headlines, Rare DiseaseTweet-
RT @CliveSvendsen: This should help the field to move forward in the complex world of C9ORF72, ALS and FTD... https://t.co/PIZRfL22l9
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Mashup Score: 7National Organization for Rare Disorders | NORD - 1 year(s) ago
NORD is dedicated to supporting education, elevating care, advancing research, and driving policy for rare diseases
Source: rarediseases.orgCategories: Latest Headlines, Rare DiseaseTweet-
The 2023 #NORDSummit has officially concluded! Thank you to our moderators, panelists, attendees, and sponsors for another successful #RareDiseases & #OrphanProducts Breakthrough Summit. Check out https://t.co/RptPreG5v1 for more events and opportunities! https://t.co/HMOKesuPaX
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Mashup Score: 2How Delta Air Lines Is Focusing On Accessibility - 1 year(s) ago
The airline is leading the way in helping make air travel more and more accessible.
Source: simpleflying.comCategories: Latest Headlines, Rare DiseaseTweet-
RT @allwheelsup: How Delta Air Lines Is Focusing On Accessibility https://t.co/yCX2r9x0O5
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https://t.co/yYBGCvWEb3