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About 893 results

ALLMedicine™ Osteopetrosis Center

Research & Reviews  337 results

Osteopetrosis: Discovery and early history of "marble bone disease".
https://doi.org/10.1016/j.bone.2023.116737
Bone Whyte MP

Mar 19th, 2023 - Discovery in 1904 of the disorder initially called "marble bones", then in 1926 more appropriately referred to as "osteopetrosis", is attributed to Heinrich E. Albers-Schönberg (1865-1921), the first radiologist. He used the new technique of Röntg...

Bone Marrow Donor Procedure
http://emedicine.medscape.com/article/1948293-overview

Mar 13th, 2023 - Background The history of stem cell transplant goes back as early as 1939, when the first documented clinical transplant was performed. In 1968, the first successful allogenic stem cell transplant was made possible followed by series of achievemen...

Bone Marrow Donor Procedure
https://emedicine.medscape.com/article/1948293-print

Mar 13th, 2023 - The history of stem cell transplant goes back as early as 1939, when the first documented clinical transplant was performed. In 1968, the first successful allogenic stem cell transplant was made possible followed by series of achievements in 1970s...

Bone Marrow Donor Procedure
https://emedicine.medscape.com/article/1948293-overview

Mar 13th, 2023 - Background The history of stem cell transplant goes back as early as 1939, when the first documented clinical transplant was performed. In 1968, the first successful allogenic stem cell transplant was made possible followed by series of achievemen...

Autosomal dominant osteopetrosis.
https://doi.org/10.1016/j.bone.2023.116723
Bone Polgreen LE, Imel EA et. al.

Mar 3rd, 2023 - Autosomal dominant osteopetrosis (ADO) is the most common form of osteopetrosis. ADO is characterized by generalized osteosclerosis along with characteristic radiographic features such as a "bone-in-bone" appearance of long bones and sclerosis of ...

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Drugs  2 results see all →

Clinicaltrials.gov  11 results

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
https://clinicaltrials.gov/ct2/show/NCT02171104

Nov 3rd, 2022 - This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with b...

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
https://clinicaltrials.gov/ct2/show/NCT04525352

Jul 13th, 2022 - This is a non-randomized Phase 1 study to evaluate the preliminary safety and efficacy of hematopoietic gene therapy consisting of autologous CD34+ enriched hematopoietic cells transduced with the lentiviral vector (LV) carrying the human TCIRG1 t...

Open Trail of γIFN for Friedreich Ataxia
https://clinicaltrials.gov/ct2/show/NCT03888664

Feb 26th, 2020 - Friedreich ataxia (FRDA) is a devastating neurodegenerative disease that affects children and young adults. Patients become progressively unable to coordinate movements and walking until severe disability ensues. Most patients develop dilated card...

Allogeneic Transplantation For Severe Osteopetrosis
https://clinicaltrials.gov/ct2/show/NCT00775931

Jul 31st, 2019 - This revised transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol and a second infusion of stem cells on day 42, 2) the mortality associated with transplant by day 100, 3) patient o...

ACTIMMUNE in Intermediate Osteopetrosis
https://clinicaltrials.gov/ct2/show/NCT02666768

Jul 2nd, 2019 - Osteopetrosis is a rare inherited metabolic bone disease characterized by impaired osteoclast function resulting in defective bone resorption and generalized high bone mass and mineral density (BMD). In patients with severe disease, this high bone...

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News  7 results

The Role of Defibrotide to Inhibit Cell Injury
https://www.onclive.com/view/the-role-of-defibrotide-to-inhibit-cell-injury

Apr 2nd, 2021 - Transcript: Sergio A. Giralt, MD: There is another study from Jeffrey Laurence, MD, where he was looking at the potential beneficial effects of defibrotide on markers of endothelial damage. And he did show that defibrotide could protect endoth...

Rare Disease Treatments Make Up Top 10 Most Costly Drugs
https://www.staging.medscape.com/viewarticle/879422

May 2nd, 2017 - UPDATED May 4, 2017 // The world's top 10 most expensive drugs are all orphan drugs for rare and ultrarare diseases, according to a new analysis. Leading that pack at number 1 is glycerol phenylbutyrate (Ravicti, Horizon Pharma), an orphan drug ap...

Rare Disease Treatments Make Up Top 10 Most Costly Drugs
https://www.medscape.com/viewarticle/879422

May 2nd, 2017 - UPDATED May 4, 2017 // The world's top 10 most expensive drugs are all orphan drugs for rare and ultrarare diseases, according to a new analysis. Leading that pack at number 1 is glycerol phenylbutyrate (Ravicti, Horizon Pharma), an orphan drug ap...

A 49-Year-Old Man With Pain at the Site of a Previous Fracture
https://reference.medscape.com/viewarticle/857143_5

Jan 14th, 2016 - On initial presentation, exclusion of other diseases that result in a similar "bright bone" appearance on radiography is important. The causes of diffuse osteosclerosis include hypervitaminosis D, hypoparathyroidism, myelofibrosis, Paget disease, ...

HLA-matched sibling transplants provide best outcomes in infantile osteopetrosis
https://www.mdedge.com/rheumatology/article/101309/transfusion-medicine/hla-matched-sibling-transplants-provide-best
Lucas Franki

Jul 18th, 2015 - Long-term survival after hematopoietic stem cell transplantation for infantile osteopetrosis was highest when grafts were taken from human leukocyte antigen (HLA)-matched siblings, according to the largest cohort of patients with the disease that.

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