Newsweek

Mom Notices Daughter Has Extreme Thirst – Then Comes Devastating Diagnosis

Holding back tears, Christine Ulrich recalls how her daughter Veda once loved dancing, drawing and babbling. Now, she can no longer speak or even hold a crayon. Three years ago, Veda was diagnosed ...
Yahoo Finance

Spruce Biosciences Receives U.S. FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB)

Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...

FDA Extends Review of DNLI's Hunter Syndrome Drug by Three Months

Denali Therapeutics faces an FDA review delay for its Hunter syndrome drug, pushing potential approval of tividenofusp alfa to April 2026.
Investing

FDA-Sonderstatus für Sanfilippo-Therapie beflügelt Aktie von Spruce Biosciences

Investing.com -- Die Aktie von Spruce Biosciences (NASDAQ:SPRB) schoss um 60 % in die Höhe, nachdem das Unternehmen bekannt gab, von der US-Arzneimittelbehörde FDA den "Breakthrough Therapy"-Status ...
Investing

Spruce Biosciences stock soars after receiving FDA Breakthrough Therapy status

Investing.com -- Spruce Biosciences (NASDAQ:SPRB) stock surged 60% after the company announced it received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for its ...

Op-Ed: The FDA’s New Direction Threatens Trump’s Innovation Agenda

President Donald Trump’s first term championed a vision of American healthcare rooted in innovation, patient choice, and ...

Spruce Shares Fall on $50M Private Placement

Shares of Spruce Biosciences were lower after the company said it had agreed to sell shares in a private placement expected to raise $50 million in gross proceeds. The stock fell 23% to $140 in ...

Ultragenyx: Rough Road Ahead Despite Growing Revenues And Catalysts

Ultragenyx (RARE) stock is down 40% since March 2024 amid ongoing financial struggles. See more insights on its growth ...
Kapitalmarktexperten

Denali Therapeutics Aktie: Kampf um die Trendwende!

Denalis aussichtsreichste Kandidaten durchlaufen derzeit entscheidende regulatorische Phasen. Der größte Hoffnungsträger ist tividenofusp alfa für die seltene Hunter-Syndrom-Erkrankung. Die ...

This Biotech Stock Soared 1,400%. Investors Are Optimistic About Its Rare-Disease Drug.

The microcap biotech stock had its best day ever after the FDA granted its rare-disease drug a breakthrough designation.
Yahoo Finance

JCR Pharmaceuticals to Present at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress

HYOGO, Japan, September 30, 2025--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR"), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic ...
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Why Spruce Biosciences Just Skyrocketed Almost 1,400%

The biotech company is working on a treatment for Sanfilippo syndrome type B, a rare and genetic disorder that affects the brain and the nervous system. Please watch the video at Investors.com - These ...