Alpha-1 Foundation Strives to Accelerate Development of New AATD Treatments
Scott Santarella spoke with Rare Disease Advisor about his new leadership position at the Alpha-1 Foundation based in Miami, Florida.
Scott Santarella spoke with Rare Disease Advisor about his new leadership position at the Alpha-1 Foundation based in Miami, Florida.
Gamida Cell is gearing up to begin sales of omidubicel from Israel to US hospitals for use in stem cell transplants, pending FDA approval of the therapy.
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Aytu BioPharma has developed an endotracheal respiratory catheter which its CEO says has “tremendous potential” in IPF, PAH, and other respiratory diseases.
Israeli biopharma giant Kamada is banking that its inhaled AAT therapy will “revolutionize” the treatment of alpha-1 antitrypsin deficiency (AATD).
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
Faron Schonfeld, a successful investment portfolio manager from Philadelphia, Pennsylvania, had for years suffered chronic coughing and sinus infections that only seemed to get worse. He was finally referred to a pulmonologist at National Jewish Health in Denver, Colorado. That’s where he got the diagnosis: Schonfeld had alpha-1 antitrypsin deficiency (AATD, or simply alpha-1) —…
Several new treatments, including potential gene therapies, are now on the horizon for people with dangerously low levels of AAT.
Under COVID-19’s lingering shadow, the National Organization of Rare Disorders (NORD) has presented its 2021 Rare Impact Awards to 24 pharmaceutical firms, researchers, politicians, and nonprofit groups for their efforts over the past year on behalf of patients with such illnesses.
Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection can cause a variety of symptoms, and people with chronic lung disease including chronic obstructive pulmonary disease (COPD), chronic liver disease especially cirrhosis, diabetes, and cardiovascular disease are among those who are more prone to develop severe infection. We now know that severe SARS-CoV-2 infection is mainly…