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.
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Omidubicel Shows Promise as Replacement for Umbilical Cord Blood in Stem Cell Transplants
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.
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The Alliance for Regenerative Medicine Fights for Better Cell, Gene Therapies
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’s Healight Device Could Help Treat Rare Pulmonary Diseases
Aytu BioPharma has developed an endotracheal respiratory catheter which its CEO says has “tremendous potential” in IPF, PAH, and other respiratory diseases.
Inhaled Therapy Could Transform AATD Treatment Landscape
Israeli biopharma giant Kamada is banking that its inhaled AAT therapy will “revolutionize” the treatment of alpha-1 antitrypsin deficiency (AATD).
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Israel Emerges as Global Powerhouse in Rare Disease Research
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.
Researchers Exploring Multiple Mechanisms of Action for Treating AATD
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) —…
Emerging Therapies, COVID-19 Highlight Alpha-1 Foundation’s Upcoming ‘Virtual Education Day’
Several new treatments, including potential gene therapies, are now on the horizon for people with dangerously low levels of AAT.
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NORD Recognizes Progress in Fight Against Rare Disease With 2021 Rare Impact Awards
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.
Examining the Links Between SARS-CoV-2 and Alpha-1 Antitrypsin Deficiency
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…
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