Zebra is a commercial stage rare disease company ... our our our our medical folks that are in there every day, educating payers around the disease, our sales reps that are in the in the offices ...

Growing up on the coast and spending time in St Lucia and Richards Bay, spotting red duikers, hippos, and crocodiles, Anban ...

Transforming Preclinical Screening with Speed, Precision, and Scalability. Explore How This Model Organism is Shaping the Next Era of Pharmaceutical Innovation. Executive Summary & Key Findings Market ...

The seven-year medical odyssey that once defined rare disease diagnosis is being rewritten by algorithms capable of identifying medical zebras in the time it takes to refresh your social media feed.

(KOIN) — A 5-year-old boy with a rare genetic disorder got an out-of-this world experience when characters from a galaxy far, far away gave him a proper Star Wars day sendoff at Portland ...

Leading the commercial launch, Tanguler is aiming for Amvuttra to become a first-line treatment due to the severity of the rare disease and the genetic medicine’s ability to knock the illness ...

To mark Rare Disease Day, pharmaphorum spoke with Paola Pozzi, partner of telethon strategy at Sofinnova Partners, about the outlook for investment in this field of research. Why is it important ...

In recognition of Rare Disease Day, Dr Kamran Iqbal, head of medical affairs and patient advocacy in global rare diseases at Chiesi UK, discussed with pharmaphorum the company’s evolving role in ...

with 13 of them dying each day on average due to insufficient organ donor supplies and systemic barriers. A New York woman's story of battling a rare disease and choosing a treatment that involved ...

Farabursen is being developed as a treatment for autosomal dominant polycystic kidney disease (ADPKD). This rare disease leads to the development of cysts in organs, mainly the kidneys.

Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company developing genetic medicines for ultra-rare and rare conditions, today announced that the U.S. Food and Drug ...

"Happy Mother’s Day! Cheers to juggling it all with joy!" Meghan wrote in her caption, sharing a rare photo of her kids Prince Archie and Princess Lilibet, who she shares with husband Prince ...