Patient Living with Pompe Disease and Participant in the COMET Clinical Trial

Alison Miles, a patient living with Pompe disease, a condition that develops when the body can’t make a protein that breaks down a complex sugar, glycogen, shares her unique diagnosis journey, the importance of self-education and patient empowerment, and the company she started, due to her mobility issues, for functional socks with grips that were fashionable.  She discusses her experience with Nexviazyme® (avalglucosidase alfa) as a patient in the COMET clinical trial.

Read More

Numinus Wellness – Psychedelic Assisted Therapies

Dr. Devon Christie, MD, is the Medical and Therapeutic Services Director with Numinus Wellness Inc., a Canadian-based company at the forefront of advancing safe, effective and innovative psychedelic-assisted therapies, and a clinical instructor with the UBC Department of Medicine, shares some insights about the therapy and why the U.S. should consider this type of therapy for those who medically qualify and what others can learn from some of Canada’s recent developments. 

Read More

Jazz Pharmaceuticals – Nothing Small About It SCLC Online Program

Abizer Gaslightwala, Vice President, Head of U.S. Hematology and Oncology at Jazz Pharmaceuticals, discusses the “Nothing Small About It” online program developed with leading cancer organizations focused exclusively on the psychosocial and emotional needs of the small cell lung cancer (SCLC) community. By creating the first and only digital hub focused exclusively on SCLC, Jazz and partners aim to inspire those living with this aggressive disease to take an active role in their health and well-being. 

Read More

New Treatment for Chronic Rhinosinusitis with Nasal Polyps

Dr. Jared Silver, MD, PhD, Allergist Immunologist & Indication Lead for Mepolizumab, US Medical Affairs Respiratory at GSK discusses the recent FDA approval of NUCALA (mepolizumab) as an add-on maintenance treatment for chronic rhinosinusitis with nasal polyps (CRSwNP) in adult patients 18 years of age and older with inadequate response to nasal corticosteroids. He explains why incorporating a biologic can be an important part of a patient’s treatment plan. 

Read More

Paragonix Technologies, Inc. – A Study On Donor Lung Transport and Preservation

Dr. Lisa Anderson, CEO, President and co-founder of Paragonix Technologies, Inc., a company delivering a new standard in organ preservation and transport, discusses a new research study called GUARDIAN-LUNG (Global Utilization And Registry Database for Improved preservAtion of doNor LUNGs) to look at lung preservation and how the way the organ is preserved and transported impact the success of the transplant. 

Read More

Sanofi Genzyme – Humanitarian Aid Program to Treat Rare Diseases

Shannon Resetich, Global Franchise Head, Rare Diseases at Sanofi Genzyme discusses the company’s Humanitarian Aid Program started 30 years ago. The program is largely focused on patients with lysosomal storage disorder (LSDs) diseases who need enzyme replacement therapies. She talks about how this humanitarian program helps to ensure patients with LSDs have access to treatment no matter where they are located or what they can afford. She also discusses how the program works effectively, the partnerships they have forged and the support that the program provides to elevate the standards for rare disease care in the communities around the world. She shares Sanofi Genzyme’s vision moving forward and why this type of program is so essential and should be part of a pharmaceutical company’s mission. 

Read More

Spinal Muscular Atrophy (SMA) Awareness – Patient Treated with Evrysdi

August is Spinal Muscular Atrophy (SMA) Awareness Month, and this year (2021) also marks the first anniversary of the FDA approval of Evrysdi (risdiplan) to treat SMA in adults and children two months and older. Here to talk about how this treatment has improved patient’s lives is Eric Arnold, a 35 year old lawyer in West Virginia who is grateful for the new treatments available to SMA patients and its transformative impact. He is joined by Dr. Karen Chen, CEO of the Spinal Muscular Atrophy Foundation who offers insights into this rare and debilitating disease and the current treatment landscape.

Read More