GENE THERAPY RARE DISEASES – Page 5 – REGENHEALTHSOLUTIONS (RHS)

PIER MARIA FORNASARI
2021-12-10

Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease.

Clinical trial planning underway to test the widely used diabetes drug metformin as a preventive treatment for the blinding eye disease Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease. Researchers from the National Eye Institute (NEI), part of

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-11-30

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A in 16 out of 18 volunteers

About 20,000 people in the U.S. live with hemophilia A. It’s a rare X-linked genetic disorder that affects predominantly males and causes their blood to clot poorly when healing wounds. For some, routine daily activities can turn into painful medical emergencies to stop internal bleeding, all because of changes in a single gene that disables

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-11-18

Gene therapy effective in children with severe Hurler’s syndrome: the treatment, developed by SR-Tiget in Milan published in NEJM

The first tests on eight children show favorable preliminary results, with an adjustment of motor and cognitive skills and improvements in some brain and joint parameters Gene therapy takes steps forward in the fight against rare diseases. Eight children with severe Hurler’s syndrome, a disease associated with reduced life expectancy, benefited from this therapy. The

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-11-07

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study published in Blood

In the last issue of Blood, Boulos et al analyze the cost-effectiveness of factor IX gene therapy in patients with severe hemophilia B, in a microsimulation Markov model. Hemophilia is an X-linked disease that affects 400000 people worldwide, of whom 15% have hemophilia B caused by deficiency of factor IX (FIX), which affected the offspring of Queen Victoria,

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-11-05

SP-101, an investigational inhaled gene therapy for cystic fibrosis, shows potential in animal study

Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD, Spirovant’s vice president of research, presented the data at the 2021 North American Cystic Fibrosis Conference (NACFC). Her

No Comments InCYSTIC FIBROSIS GENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-10-27

Gene Therapies against Rare diseases Boosted by NIH, FDA and Industry Alliance

The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Bespoke

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-10-23

Gene Therapy Up to Date

After numerous setbacks at the turn of the century, gene therapy is treating diseases ranging from neuromuscular disorders to cancer to blindness. The success is often qualified, however. Some of these therapies have proved effective at alleviating disease but come with a high price tag and other accessibility issues: Even when people know that a

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-10-23

Gene Therapy Shows Early Promise as Angelman Syndrome Treatment

The new therapy was generally well-tolerated and prevented key signs of the condition in animal models. Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities. Angelman

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-06-05

Gene therapy appears more cost effective than current treatments for severe hemophilia B

St. Jude Children’s Research Hospital provides the first gene therapy manufacturing and distribution cost report for hemophilia B. A St. Jude Children’s Research Hospital analysis found a major gap between the cost to manufacture and distribute hemophilia B gene therapy and the $2 million-plus price reportedly under consideration for hemophilia gene therapy now in development. The study appeared

No Comments InGENE THERAPY RARE DISEASES

PIER MARIA FORNASARI
2021-05-21

Gene therapy offers potential cure to children born with immunodeficiency due to adenosine deaminase deficiency.

Study shows treatment developed by international team restored immune function in more than 95% of patients in three clinical trials An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited

No Comments InGENE THERAPY RARE DISEASES

REGENERATIVE AND PERSONALISED MEDICINE

DIGITAL HEALTHCARE

Category GENE THERAPY RARE DISEASES

Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease.

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A in 16 out of 18 volunteers

Gene therapy effective in children with severe Hurler’s syndrome: the treatment, developed by SR-Tiget in Milan published in NEJM

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study published in Blood

SP-101, an investigational inhaled gene therapy for cystic fibrosis, shows potential in animal study

Gene Therapies against Rare diseases Boosted by NIH, FDA and Industry Alliance

Gene Therapy Up to Date

Gene Therapy Shows Early Promise as Angelman Syndrome Treatment

Gene therapy appears more cost effective than current treatments for severe hemophilia B

Gene therapy offers potential cure to children born with immunodeficiency due to adenosine deaminase deficiency.