rare

BlackfinBio Announces FDA Clearance to Initiate Phase 1/2 Clinical Trial for Novel AAV Gene Therapy BFB-101 to Treat the Rare Neurological Disease Hereditary Spastic Paraplegia, Type 47 (SPG47)BlackfinBio Announces FDA Clearance to Initiate Phase 1/2 Clinical Trial for Novel AAV Gene Therapy BFB-101 to Treat the Rare Neurological Disease Hereditary Spastic Paraplegia, Type 47 (SPG47)

BlackfinBio Announces FDA Clearance to Initiate Phase 1/2 Clinical Trial for Novel AAV Gene Therapy BFB-101 to Treat the Rare Neurological Disease Hereditary Spastic Paraplegia, Type 47 (SPG47)

April 28, 2025 04:00 ET  | Source: BlackfinBio CHESHIRE, United Kingdom, April 28, 2025 (GLOBE NEWSWIRE) -- BlackfinBio Ltd, a…

3 weeks ago
Rare Indian Skimmer Sighting at Haiderpur Wetland by The Art of Living Sparks HopeRare Indian Skimmer Sighting at Haiderpur Wetland by The Art of Living Sparks Hope

Rare Indian Skimmer Sighting at Haiderpur Wetland by The Art of Living Sparks Hope

BENGALURU, India, April 16, 2025 /PRNewswire/ -- In a landmark moment for Indian conservation, a rare and thrilling sighting of…

1 month ago
Norgine strengthens rare disease portfolio with acquisition of TheraviaNorgine strengthens rare disease portfolio with acquisition of Theravia

Norgine strengthens rare disease portfolio with acquisition of Theravia

Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio…

1 month ago

Rare loss-of-function variants in HECTD2 and AKAP11 confer a risk of bipolar disorder

Scientists at deCODE genetics, a subsidiary of Amgen, reveal today in Nature Genetics associations between rare loss-of-function variants in two genes…

2 months ago
Regeneron Science Talent Search 2025 Awards More Than $1.8 Million to High School Seniors for Innovative Scientific Research on Classifying Objects in Space, Treating a Rare Muscle Disease and Solving a Long-Standing Math ProblemRegeneron Science Talent Search 2025 Awards More Than $1.8 Million to High School Seniors for Innovative Scientific Research on Classifying Objects in Space, Treating a Rare Muscle Disease and Solving a Long-Standing Math Problem

Regeneron Science Talent Search 2025 Awards More Than $1.8 Million to High School Seniors for Innovative Scientific Research on Classifying Objects in Space, Treating a Rare Muscle Disease and Solving a Long-Standing Math Problem

TARRYTOWN, N.Y. and WASHINGTON, March 11, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Society for Science (the…

2 months ago

NYSE CONTENT ADVISORY: PRE-MARKET UPDATE + RARE DISEASE DAY AWARENESS

NEW YORK, Feb. 28, 2025 /PRNewswire/ -- The New York Stock Exchange (NYSE) is proud to offer a daily pre-market…

3 months ago
Bhandup West Embraces Healthy Sundays with Arkade Developers Happy Streets at Arkade RareBhandup West Embraces Healthy Sundays with Arkade Developers Happy Streets at Arkade Rare

Bhandup West Embraces Healthy Sundays with Arkade Developers Happy Streets at Arkade Rare

~  In the last 5 weekends, the event has witnessed a crowd of more than approximately 5000 people ~  Introduced…

3 months ago
The Avestagenome Project and TIGS Sign Strategic Alliance to Advance Research in Rare Genetic DisordersThe Avestagenome Project and TIGS Sign Strategic Alliance to Advance Research in Rare Genetic Disorders

The Avestagenome Project and TIGS Sign Strategic Alliance to Advance Research in Rare Genetic Disorders

BENGALURU, India, Feb. 24, 2025 /PRNewswire/ -- The Avestagenome Project® International Pvt. Ltd. (AGENOME) & Avesthagen Limited, in partnership with the…

3 months ago
Egetis Therapeutics to Feature MCT8 Deficiency on Behind the Mystery Airing on Lifetime in Honor of Rare Disease DayEgetis Therapeutics to Feature MCT8 Deficiency on Behind the Mystery Airing on Lifetime in Honor of Rare Disease Day

Egetis Therapeutics to Feature MCT8 Deficiency on Behind the Mystery Airing on Lifetime in Honor of Rare Disease Day

STOCKHOLM, Feb. 20, 2025 (GLOBE NEWSWIRE) -- Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), an innovative…

3 months ago
Kavalan Completes Its ‘Sherry Family’ With Rare Palo Cortado CaskKavalan Completes Its ‘Sherry Family’ With Rare Palo Cortado Cask

Kavalan Completes Its ‘Sherry Family’ With Rare Palo Cortado Cask

Exceptional whisky delivers hints of vanilla fruitcake, fragrant cardamom, and orange pound cake TAIPEI, Feb. 18, 2025 /PRNewswire/ -- Kavalan…

3 months ago