ChallengesExperts share their insights in diagnosing and managing rare conditions
- The Success of the Orphan Drug Act
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses the success of the Orphan Drug Act (ODA).
- Preliminary Results from Phase 2 Trial in Patients with Charcot-Marie-Tooth Disease Show Promise
There seems to be some good news on the horizon for individuals with Charcot-Marie-Tooth (CMT) disease. Acceleron Pharma announced positive preliminary results from Part 1 of the Phase 2 clinical trial with ACE-083 in patients with CMT disease at the Peripheral Nerve Society (PNS) annual meeting.
- Considering Rare Diseases for Challenging Cases
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses how physicians need to consider rare diseases for some of their more challenging diagnositic cases.
- FDA Approves Tibsovo, First IDH1 Inhibitor for Acute Myeloid Leukemia
The U.S. Food and Drug Administration (FDA) approved Tibsovo (ivosidenib) tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation.
- Challenges of the Health Care System Supporting Innovative Drugs
Chris Garabedian discusses the challenges of our health care syste3m supporting the many emerging and innovative therapies entering the market.
- The Right to Try Act
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses Right To Try, which is aimed at allowing terminally ill Americans to try medicines that have passed Phase 1 of the FDA approval process and remain in clinical trials but are not yet on pharmacy shelves.
- Daniel de Boer Discusses His Company’s Focus on Usher Syndrome
Daniel de Boer, Founder and CEO of ProQR, discusses his company’s clinical program focusing on Usher syndrome is a rare genetic disease that is the leading cause of combined deafness and blindness.
- Scioto Biosciences Focus on Microbiome Therapeutics
Dr. Joe Trebley, Co-founder and CEO of Scioto Biosciences, discusses his company’s focus on developing a novel probiotic delivery platform that provides enhanced function and persistent probiotic benefits to patients.
- Dilanubicel Receives Orphan Drug Status for Reduction of Morbidity and Mortality Associated With HSCT
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to dilanubicel (Nohla Therapeutics) for the reduction of morbidity and mortality associated with hematopoietic stem cell transplant (HSCT).
- Scioto Biosciences: Focused on Reducing Necrotizing Enterocolitis
Jim Schulz, Co-founder and Chief Financial Officer of Scioto Biosciences, discusses his company’s focus on developing therapies devoted to having a impact on the delivery of microbiome therapeutics with the goal of reducing the incidence of necrotizing enterocolitis (NEC).