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737,750 Results for "rare disease therapeutics inc".
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Rare diseases
How SpringWorks Took Pfizer’s Rare Disease Ghost Drug and Got It to Patients
SpringWorks Therapeutics sprung out of Pfizer’s storeroom, when a rare disease advocacy group pushed to keep a program for neurofibromatosis alive. This method could work for “every rare disease under the sun,” advocates say.
February 26, 2025
·
5 min read
·
Annalee Armstrong
FDA
Makary Discusses Expedited Rare Disease Approvals Pathway, ‘Public Distrust’ in New Interview
In an interview with former
Fox News
journalist Megyn Kelly, FDA Commissioner Marty Makary introduced a new mechanism-driven pathway that could be leveraged by rare disease therapies while saying that autism could potentially be driven by certain environmental factors.
April 21, 2025
·
2 min read
·
Tristan Manalac
Press Releases
Odylia Therapeutics drives commitment to rare disease drug development through Rare Disease Day event
April 11, 2025
·
4 min read
Funding
AIRNA Hits $155M Series B Haul To Advance RNA Editor for Rare Genetic Disease
AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 antitrypsin deficiency.
April 1, 2025
·
2 min read
·
Tristan Manalac
Rare diseases
Glycomine Gets $115M Series C for Rare Disease, Aims for Phase II Launch Mid-Year
The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a rare disease that affects the entire body and produces a wide range of symptoms.
April 16, 2025
·
2 min read
·
Dan Samorodnitsky
Rare diseases
How To Mine Biopharma’s IP Storeroom for Rare Disease Drugs, Just Like SpringWorks
SpringWorks Therapeutics is the perfect case study for rescuing a discontinued assets. It’s time to repeat the process for every rare disease, experts say.
February 26, 2025
·
5 min read
·
Annalee Armstrong
Rare diseases
Rare Disease Biotechs Left in a Lurch as Congress Fails To Renew Priority Review Program
Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma.
March 5, 2025
·
9 min read
·
Annalee Armstrong
Approvals
Mirum Establishes Liver Expertise With First FDA Nod for ‘Very Rare’ Disease
Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
February 24, 2025
·
2 min read
·
Tristan Manalac
Press Releases
Norgine strengthens rare disease portfolio with acquisition of Theravia
April 16, 2025
·
7 min read
Press Releases
BioMark Diagnostics Underscores Commitment to Rare Disease Research on Rare Disease Day 2025
BioMark is pioneering liquid biopsy solutions for challenging conditions and championing equity in diagnosis
February 28, 2025
·
3 min read
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