Denali has entered a $275m royalty funding agreement with Royalty Pharma based on future net sales of tividenofusp alfa ...

The FDA has placed a hold on Denali Therapeutics’ plans to launch a phase 1 rare disease trial, citing concerns about immune ...

Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 ...

Denali Therapeutics Inc. DNLI has announced a funding agreement with Royalty Pharma plc RPRX for $275 million. The stock ...

In return, Royalty will receive a 9.25% royalty on worldwide sales of tividenofusp alfa from Denali until it reaches three times the amount Royalty has handed to Denali—or, alternatively, 2.5 times ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...

FDA reports rare neutralizing antibodies, including a pediatric death, in patients treated with Takeda's Adzynma for congenital TTP.

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...

It’s a big day for Sanofi as the pharmaceutical giant welcomes two European drug approvals, both for rare enzyme deficiencies. One regulatory nod ushers in the first treatment in Europe for a ...

Neurocognitive function, such as early IQ, was measured with the Mullen Scales of Early Learning or Bayley Scales of Infant and Toddler Development 3rd Edition. The following article features coverage ...

The US Food and Drug Administration (FDA) has approved a new enzyme replacement therapy, avalglucosidase alfa (Nexviazyme, Genzyme), for patients aged 1 year and older with late-onset Pompe disease.