CIRM blog

Meeting the scientists who are turning their daughter’s cells into a research tool

There’s nothing like a face-to-face meeting to really get to know someone. And when the life of someone you love is in the hands of that person, then it’s a meeting that comes packed with emotion and importance. Last week Gay and Steve Grossman got to meet the people who are working with their daughter Lilly’s stem cells. Lilly was …

CIRM

Patient Advocate Leads Drive to Add Rare Diseases to CIRM’s Stem Cell Bank

November 1, 2016 Oakland, CA – Christina Waters is a rare kind of person: creative, compassionate, and committed to creating change. Now she is using those rare talents to help children battling rare diseases. She is helping add blood and skin samples from these children to be turned into stem cells and used as a resource for researchers to better understand …

Rising costs may not be a result of orphan drugs

Published Online: Sep 12,2016 Laurie Toich, Assistant Editor From Daraprim’s price rising 5556% by Turing Pharmaceuticals under the leadership of Martin Shkreli, to the most recent pricing scandal with Mylan’s EpiPen, Americans have been faced with high out-of-pocket costs for many prescription drugs. Government institutions, such as the US Centers for Medicare and Medicaid, have begun crafting novel ways to reduce …

Rare diseases

Sharing the mission to conquer rare diseases

Closer collaboration between pharmaceutical companies and patient organizations will accelerate drug development At an ever-quickening pace, biopharmaceutical companies and patient advocacy groups are banding together to hasten development of new drugs for rare diseases. Advocacy groups rely on their partners for financial and scientific support while providing access to repositories of patient data – the key to understanding rare diseases …

BioPharma Mexico is where experts like Shire Mexico meet to network, learn from each other, and discuss the latest developments in biopharma.

What Does Shire Mexico Have to Say About Orphan Drugs?

We’re excited to announce that Raul Vivar, General Director of Shire Mexico, will be speaking at this year’s BioPharma Mexico. Raul’s presentation is titled “Accessing public institutions: A success story changing the lives of lysosomal disease patients”, and will take place at 4:05 pm on Wednesday, November 5th. Don’t miss it! Download our interview with Raul here During his presentation, …

Can Gold Fight Cancer?

Exciting developments have gripped the cancer research space as a result of a “Trojan horse” treatment for Glioblastoma Multiforme, an extremely aggressive form of brain cancer which sees a devastating survival rate at just six in every 100 patients making it past the 5-year mark. This form of brain cancer is notorious as adults rarely survive past the first months of diagnosis. …

The World Orphan Drug Congress USA is where pharma, government, and patient groups gather to network, learn from each other, and discuss the latest orphan drug and rare disease strategies and technologies.

The Role of Specialty Pharmacy in Orphan Drug Therapy

Orsini Healthcare is a leading national specialty pharmacy focused on the delivery of specialty medications to patients with rare conditions. Their disease-specific clinical CareTeam programs, commitment to high-touch customer service, and our patient centered approach ensures satisfaction and adherence to treatment. Orsini Healthcare will be exhibiting at this year’s World Orphan Drug Congress USA, and we were lucky enough to …

The World Orphan Drug Congress USA is where the rare disease industry comes together to network, learn from each other, and discuss the latest orphan drug developments.

Pat Furlong of Parents Project Muscular Dystrophy on the World Orphan Drug Congress USA

One of the advocacy leaders in the rare disease community is Pat Furlong, President of Parents Project Muscular Dystrophy (PPMD). This April, Ms Furlong will be speaking at the upcoming World Orphan Drug Congress USA in Washington D.C. In this exclusive interview with Rare Disease Report, Ms Furlong explains why she thinks this is one of the most important rare …

The World Orphan Drug Congress is where the orphan drug industry goes to network, learn from each other, and discuss the latest strategies and technology.

The Role of Big Pharma in Orphan Drug Development and Commercialization

Orphan drug development is often perceived as being done by small biotech companies. However, most big pharmaceutical companies are heavily invested in the rare disease community. In this exclusive interview with Rare Disease Report, Bert Bruce, Vice President of Rare Disease Commercial Development at Pfizer Inc, previews his presentation at the upcoming World Orphan Drug Congress USA 2014 being held …

Eldest son of actress Audrey Hepburn named as Rare Disease Day Ambassador 2014

Rare Disease Day 2014 is just 3 weeks away and we understand from EURODIS – the voice of rare disease patients in Europe – that Sean Hepburn Ferrer has been named the Rare Disease Day Ambassador 2014. For the full article, read http://www.rarediseaseday.org/article/sean-hepburn-ferrer-rare-disease-day-2014-ambassador Sean Hepburn Ferrer is the eldest son of the late, beloved Audrey Hepburn, actress and dedicated philanthropist, …